"Mol Ther Methods Clin Dev"[ Journal] - Search Results

Year	Number of Results
2014	68
2015	60
2016	104
2017	101
2018	135
2019	175
2020	289
2021	228
2022	185
2023	211
2024	68

1

Durable transgene expression and efficient re-administration after rAAV2.5T-mediated fCFTRΔR gene delivery to adult ferret lungs.

Tang Y, Ebadi M, Lei J, Feng Z, Fakhari S, Wu P, Smith MD, Limberis MP, Kolbeck R, Excoffon KJ, Yan Z, Engelhardt JF. Tang Y, et al. Mol Ther Methods Clin Dev. 2024 Mar 28;32(2):101244. doi: 10.1016/j.omtm.2024.101244. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38638546 Free PMC article.

2

Development of adenoviral vectors that transduce Purkinje cells and other cerebellar cell-types in the cerebellum of a humanized mouse model.

Kul E, Okoroafor U, Dougherty A, Palkovic L, Li H, Valiño-Ramos P, Aberman L, Young SM Jr. Kul E, et al. Mol Ther Methods Clin Dev. 2024 Mar 26;32(2):101243. doi: 10.1016/j.omtm.2024.101243. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38605812 Free PMC article.

3

Subretinal AAV delivery of RNAi-therapeutics targeting VEGFA reduces choroidal neovascularization in a large animal model.

Haldrup SH, Fabian-Jessing BK, Jakobsen TS, Lindholm AB, Adsersen RL, Aagaard L, Bek T, Askou AL, Corydon TJ. Haldrup SH, et al. Mol Ther Methods Clin Dev. 2024 Mar 22;32(2):101242. doi: 10.1016/j.omtm.2024.101242. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38605811 Free PMC article.

4

A toxicology study of Csf2ra complementation and pulmonary macrophage transplantation therapy of hereditary PAP in mice.

Arumugam P, Carey BC, Wikenheiser-Brokamp KA, Krischer J, Wessendarp M, Shima K, Chalk C, Stock J, Ma Y, Black D, Imbrogno M, Collins M, Kalenda Yombo DJ, Sakthivel H, Suzuki T, Lutzko C, Cancelas JA, Adams M, Hoskins E, Lowe-Daniels D, Reeves L, Kaiser A, Trapnell BC. Arumugam P, et al. Mol Ther Methods Clin Dev. 2024 Feb 17;32(2):101213. doi: 10.1016/j.omtm.2024.101213. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38596536 Free PMC article.

5

Combining CRISPR-Cas-mediated terminal resolution with a novel genetic workflow to achieve high-diversity adenoviral libraries.

Fischer J, Fedotova A, Jaki L, Sallard E, Erhardt A, Fuchs J, Ruzsics Z. Fischer J, et al. Mol Ther Methods Clin Dev. 2024 Mar 18;32(2):101241. doi: 10.1016/j.omtm.2024.101241. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38585687 Free PMC article.

6

mtDNA analysis using Mitopore.

Dobner J, Nguyen T, Pavez-Giani MG, Cyganek L, Distelmaier F, Krutmann J, Prigione A, Rossi A. Dobner J, et al. Mol Ther Methods Clin Dev. 2024 Mar 12;32(2):101231. doi: 10.1016/j.omtm.2024.101231. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38572068 Free PMC article.

7

Protein phosphatase 2A anchoring disruptor gene therapy for familial dilated cardiomyopathy.

Li X, Li J, Samuelsson AM, Thakur H, Kapiloff MS. Li X, et al. Mol Ther Methods Clin Dev. 2024 Mar 11;32(2):101233. doi: 10.1016/j.omtm.2024.101233. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38572067 Free PMC article.

8

Promise of gene therapy for congenital neurologic disease due to GPI deficiency.

Babushok DV, Sabatino DE. Babushok DV, et al. Mol Ther Methods Clin Dev. 2024 Mar 25;32(2):101240. doi: 10.1016/j.omtm.2024.101240. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38572066 Free PMC article. No abstract available.

9

Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection.

Liu H, Zhang Y, Yip M, Ren L, Liang J, Chen X, Liu N, Du A, Wang J, Chang H, Oh H, Zhou C, Xing R, Xu M, Guo P, Gessler D, Xie J, Tai PWL, Gao G, Wang D. Liu H, et al. Mol Ther Methods Clin Dev. 2024 Mar 12;32(2):101230. doi: 10.1016/j.omtm.2024.101230. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38558570 Free PMC article.

10

Novel AAV variants with improved tropism for human Schwann cells.

Drouyer M, Chu TH, Labit E, Haase F, Navarro RG, Nazareth D, Rosin N, Merjane J, Scott S, Cabanes-Creus M, Westhaus A, Zhu E, Midha R, Alexander IE, Biernaskie J, Ginn SL, Lisowski L. Drouyer M, et al. Mol Ther Methods Clin Dev. 2024 Mar 11;32(2):101234. doi: 10.1016/j.omtm.2024.101234. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38558569 Free PMC article.

11

AAV-delivered hepato-adrenal cooperativity in steroidogenesis: Implications for gene therapy for congenital adrenal hyperplasia.

Graves LE, van Dijk EB, Zhu E, Koyyalamudi S, Wotton T, Sung D, Srinivasan S, Ginn SL, Alexander IE. Graves LE, et al. Mol Ther Methods Clin Dev. 2024 Mar 12;32(2):101232. doi: 10.1016/j.omtm.2024.101232. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38558568 Free PMC article.

12

Embryo and fetal gene editing: Technical challenges and progress toward clinical applications.

Mattar CNZ, Chew WL, Lai PS. Mattar CNZ, et al. Mol Ther Methods Clin Dev. 2024 Mar 4;32(2):101229. doi: 10.1016/j.omtm.2024.101229. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38533521 Free PMC article. Review.

13

A robust and flexible baculovirus-insect cell system for AAV vector production with improved yield, capsid ratios and potency.

Marwidi Y, Nguyen HB, Santos D, Wangzor T, Bhardwaj S, Ernie G, Prawdzik G, Lew G, Shivak D, Trias M, Padilla J, Tran H, Meyer K, Surosky R, Ward AM. Marwidi Y, et al. Mol Ther Methods Clin Dev. 2024 Mar 4;32(2):101228. doi: 10.1016/j.omtm.2024.101228. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38524756 Free PMC article.

14

Strengthening health systems for access to gene therapy in rare genetic disorders.

Bhatia S, Le Cam Y, Carrion J, Diamond L, Fennessy P, Gassman S, Gutzwiller F, Kagan S, Pankevich D, Young Maloney J, Mahadev N, Schulz M, Wong-Rieger D, Morgese P. Bhatia S, et al. Mol Ther Methods Clin Dev. 2024 Mar 13;32(2):101220. doi: 10.1016/j.omtm.2024.101220. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38516694 Free PMC article. No abstract available.

15

Endovascular transplantation of mRNA-enhanced mesenchymal stromal cells results in superior therapeutic protein expression in swine heart.

Al-Saadi J, Waldén M, Sandell M, Sohlmér J, Grankvist R, Friberger I, Andersson A, Carlsten M, Chien K, Lundberg J, Witman N, Holmin S. Al-Saadi J, et al. Mol Ther Methods Clin Dev. 2024 Feb 27;32(2):101225. doi: 10.1016/j.omtm.2024.101225. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38516693 Free PMC article.

16

Peptide-encoding gene transfer to modulate intracellular protein-protein interactions.

Taya T, Kami D, Teruyama F, Matoba S, Gojo S. Taya T, et al. Mol Ther Methods Clin Dev. 2024 Feb 28;32(2):101226. doi: 10.1016/j.omtm.2024.101226. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38516692 Free PMC article.

17

Manufacturing DNA in E. coli yields higher-fidelity DNA than in vitro enzymatic synthesis.

Hersch SJ, Chandrasekaran S, Lam J, Nafissi N, Slavcev RA. Hersch SJ, et al. Mol Ther Methods Clin Dev. 2024 Feb 28;32(2):101227. doi: 10.1016/j.omtm.2024.101227. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38516691 Free PMC article.

18

Automated manufacture of ΔNPM1 TCR-engineered T cells for AML therapy.

Yonezawa Ogusuku IE, Herbel V, Lennartz S, Brandes C, Argiro E, Fabian C, Hauck C, Hoogstraten C, Veld S, Hageman L, Teppert K, Koutsoumpli G, Griffioen M, Mockel-Tenbrinck N, Schaser T, de Groot R, Johnston ICD, Lock D. Yonezawa Ogusuku IE, et al. Mol Ther Methods Clin Dev. 2024 Feb 27;32(2):101224. doi: 10.1016/j.omtm.2024.101224. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38516690 Free PMC article.

19

Preexisting antibody assays for gene therapy: Considerations on patient selection cutoffs and companion diagnostic requirements.

Braun M, Lange C, Schatz P, Long B, Stanta J, Gorovits B, Tarcsa E, Jawa V, Yang TY, Lembke W, Miller N, McBlane F, Christodoulou L, Yuill D, Milton M. Braun M, et al. Mol Ther Methods Clin Dev. 2024 Feb 20;32(1):101217. doi: 10.1016/j.omtm.2024.101217. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38496304 Free PMC article. Review.

20

Glutaredoxin-1 modulates the NF-κB signaling pathway to activate inducible nitric oxide synthase in experimental necrotizing enterocolitis.

Zhang Y, Yan M, Xia Y, Yue Y, Wang S, Hu Y, Lai G, Wu Q, Liu Q, Ding X, Guo C. Zhang Y, et al. Mol Ther Methods Clin Dev. 2024 Feb 19;32(1):101214. doi: 10.1016/j.omtm.2024.101214. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38496303 Free PMC article.

21

Non-canonical capsid engineering highlights new possibilities for AAV vectorology.

Zhang Z, Counsell JR. Zhang Z, et al. Mol Ther Methods Clin Dev. 2024 Mar 6;32(1):101221. doi: 10.1016/j.omtm.2024.101221. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38487107 Free PMC article. No abstract available.

22

Thorough molecular configuration analysis of noncanonical AAV genomes in AAV vector preparations.

Zhang J, Yu X, Chrzanowski M, Tian J, Pouchnik D, Guo P, Herzog RW, Xiao W. Zhang J, et al. Mol Ther Methods Clin Dev. 2024 Feb 19;32(1):101215. doi: 10.1016/j.omtm.2024.101215. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38463141 Free PMC article.

23

Optimizing lentiviral genomic integrations to cure beta-thalassemia: The least required for success?

Rivella S. Rivella S. Mol Ther Methods Clin Dev. 2024 Mar 5;32(1):101222. doi: 10.1016/j.omtm.2024.101222. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38463140 Free PMC article. No abstract available.

24

Optimization of anti-CD19 CAR T cell production for treatment of patients with chronic lymphocytic leukemia.

Amatya C, Weissler KA, Fellowes V, Lam N, Cutmore LC, Natrakul DA, Highfill SL, Kochenderfer JN. Amatya C, et al. Mol Ther Methods Clin Dev. 2024 Feb 13;32(1):101212. doi: 10.1016/j.omtm.2024.101212. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38455264 Free PMC article.

25

The very stable lentiviral vector.

Binder GK, Chen CC. Binder GK, et al. Mol Ther Methods Clin Dev. 2024 Mar 5;32(1):101223. doi: 10.1016/j.omtm.2024.101223. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38455263 Free PMC article. No abstract available.

26

Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques.

Blackwood M, Gruntman AM, Tang Q, Pires-Ferreira D, Reil D, Kondratov O, Marsic D, Zolotukhin S, Gernoux G, Keeler AM, Mueller C, Flotte TR. Blackwood M, et al. Mol Ther Methods Clin Dev. 2024 Jan 30;32(1):101200. doi: 10.1016/j.omtm.2024.101200. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38445045 Free PMC article.

27

A positive take on negative selection for CAR-T manufacturing.

Orentas RJ. Orentas RJ. Mol Ther Methods Clin Dev. 2024 Mar 1;32(1):101218. doi: 10.1016/j.omtm.2024.101218. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38445044 Free PMC article. No abstract available.

28

B cell focused transient immune suppression protocol for efficient AAV readministration to the liver.

Rana J, Herzog RW, Muñoz-Melero M, Yamada K, Kumar SRP, Lam AK, Markusic DM, Duan D, Terhorst C, Byrne BJ, Corti M, Biswas M. Rana J, et al. Mol Ther Methods Clin Dev. 2024 Feb 20;32(1):101216. doi: 10.1016/j.omtm.2024.101216. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38440160 Free PMC article.

29

Extracellular vesicles ameliorates sleep deprivation induced anxiety-like behavior and cognitive impairment in mice.

Lin Y, Kang Z, Su C, Li S, Xie W. Lin Y, et al. Mol Ther Methods Clin Dev. 2024 Feb 5;32(1):101207. doi: 10.1016/j.omtm.2024.101207. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38435131 Free PMC article.

30

Rationally engineered novel AAV capsids for intra-articular gene delivery.

Li W, Feng SL, Herrschaft L, Samulski RJ, Li C. Li W, et al. Mol Ther Methods Clin Dev. 2024 Feb 14;32(1):101211. doi: 10.1016/j.omtm.2024.101211. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38435130 Free PMC article.

31

Genome editing in clinical practice: A model study for next-gen hematopoietic cell transplants in hematologic malignancies.

Derigs P, Müller-Tidow C. Derigs P, et al. Mol Ther Methods Clin Dev. 2024 Feb 26;32(1):101210. doi: 10.1016/j.omtm.2024.101210. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38435129 Free PMC article. No abstract available.

32

Continuous manufacturing of lentiviral vectors using a stable producer cell line in a fixed-bed bioreactor.

Stibbs DJ, Silva Couto P, Takeuchi Y, Rafiq QA, Jackson NB, Rayat ACME. Stibbs DJ, et al. Mol Ther Methods Clin Dev. 2024 Feb 9;32(1):101209. doi: 10.1016/j.omtm.2024.101209. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38435128 Free PMC article.

33

Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome.

Pille M, Avila JM, Park SH, Le CQ, Xue H, Haerynck F, Saxena L, Lee C, Shpall EJ, Bao G, Vandekerckhove B, Davis BR. Pille M, et al. Mol Ther Methods Clin Dev. 2024 Feb 6;32(1):101208. doi: 10.1016/j.omtm.2024.101208. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38414825 Free PMC article.

34

Gesicles packaging dCas9-VPR ribonucleoprotein complexes can combine with vorinostat and promote HIV proviral transcription.

Fisher MA, Chaudhry W, Campbell LA. Fisher MA, et al. Mol Ther Methods Clin Dev. 2024 Feb 2;32(1):101203. doi: 10.1016/j.omtm.2024.101203. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38390557 Free PMC article.

35

Large-scale generation of IL-12 secreting macrophages from human pluripotent stem cells for cancer therapy.

Kang B, Xing Q, Huang Y, Lin H, Peng J, Zhang Z, Wang M, Guo X, Hu X, Wang S, Wang J, Gao M, Zhu Y, Pan G. Kang B, et al. Mol Ther Methods Clin Dev. 2024 Feb 2;32(1):101204. doi: 10.1016/j.omtm.2024.101204. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38390556 Free PMC article.

36

A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR.

Zhang J, Frabutt DA, Chrzanowski M, Li N, Miller LM, Tian J, Mulcrone PL, Lam AK, Draper BE, Jarrold MF, Herzog RW, Xiao W. Zhang J, et al. Mol Ther Methods Clin Dev. 2024 Feb 3;32(1):101206. doi: 10.1016/j.omtm.2024.101206. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38390555 Free PMC article.

37

All-in-one IQ toggle switches with high versatilities for fine-tuning of transgene expression in mammalian cells and tissues.

Hong J, Sohn KC, Park HW, Jeon H, Ju E, Lee JG, Lee JS, Rho J, Hur GM, Ro H. Hong J, et al. Mol Ther Methods Clin Dev. 2024 Feb 2;32(1):101202. doi: 10.1016/j.omtm.2024.101202. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38374964 Free PMC article.

38

AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life.

Doshi BS, Samelson-Jones BJ, Nichols TC, Merricks EP, Siner JL, French RA, Lee BJ, Arruda VR, Callan MB. Doshi BS, et al. Mol Ther Methods Clin Dev. 2024 Feb 2;32(1):101205. doi: 10.1016/j.omtm.2024.101205. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38374963 Free PMC article.

39

Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice.

Smith MC, Belur LR, Karlen AD, Erlanson O, Furcich J, Lund TC, Seelig D, Kitto KF, Fairbanks CA, Kim KH, Buss N, McIvor RS. Smith MC, et al. Mol Ther Methods Clin Dev. 2024 Jan 30;32(1):101201. doi: 10.1016/j.omtm.2024.101201. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38374962 Free PMC article.

40

First use of adeno-associated viruses in the human inner ear.

Landegger LD. Landegger LD. Mol Ther Methods Clin Dev. 2024 Feb 10;32(1):101197. doi: 10.1016/j.omtm.2024.101197. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38371610 Free PMC article. No abstract available.

41

A gene silencing-based approach to tackle fatty liver disease.

Strnad P, Schrader C, Brunetti-Pierri N. Strnad P, et al. Mol Ther Methods Clin Dev. 2024 Feb 9;32(1):101198. doi: 10.1016/j.omtm.2024.101198. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38371609 Free PMC article. No abstract available.

42

Navigating toward gene therapy in Marfan syndrome: A hope for halting aortic aneurysm.

Egea G. Egea G. Mol Ther Methods Clin Dev. 2024 Feb 8;32(1):101196. doi: 10.1016/j.omtm.2024.101196. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38357700 Free PMC article. No abstract available.

43

Erratum: A novel FOXP3 knockout-humanized mouse model for pre-clinical safety and efficacy evaluation of Treg-like cell products.

Sato Y, Nathan A, Shipp S, Wright JF, Tate KM, Wani P, Roncarolo MG, Bacchetta R. Sato Y, et al. Mol Ther Methods Clin Dev. 2024 Feb 5;32(1):101199. doi: 10.1016/j.omtm.2024.101199. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38352272 Free PMC article.

44

Whole-body galactose oxidation as a robust functional assay to assess the efficacy of gene-based therapies in a mouse model of Galactosemia.

Balakrishnan B, Yan X, McCue MD, Bellagamba O, Guo A, Winkler F, Thall J, Crawford L, Dimen R, Chen S, McEnaney S, Wu Y, Zimmer M, Sarkis J, Martini PGV, Finn PF, Lai K. Balakrishnan B, et al. Mol Ther Methods Clin Dev. 2024 Jan 17;32(1):101191. doi: 10.1016/j.omtm.2024.101191. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38352271 Free PMC article.

45

AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich's ataxia.

Chang JC, Ryan MR, Stark MC, Liu S, Purushothaman P, Bolan F, Johnson CA, Champe M, Meng H, Lawlor MW, Halawani S, Ngaba LV, Lynch DR, Davis C, Gonzalo-Gil E, Lutz C, Urbinati F, Medicherla B, Fonck C. Chang JC, et al. Mol Ther Methods Clin Dev. 2024 Jan 22;32(1):101193. doi: 10.1016/j.omtm.2024.101193. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38352270 Free PMC article.

46

Liter-scale manufacturing of shelf-stable plasmid DNA/PEI transfection particles for viral vector production.

Hu Y, Eder BA, Lin J, Li S, Zhu Y, Wang TH, Guo T, Mao HQ. Hu Y, et al. Mol Ther Methods Clin Dev. 2024 Jan 22;32(1):101194. doi: 10.1016/j.omtm.2024.101194. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38352269 Free PMC article.

47

Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency.

Mével M, Pichard V, Bouzelha M, Alvarez-Dorta D, Lalys PA, Provost N, Allais M, Mendes A, Landagaray E, Ducloyer JB, Toublanc E, Galy A, Brument N, Lefevre GM, Gouin SG, Isiegas C, Le Meur G, Cronin T, Le Guiner C, Weber M, Moullier P, Ayuso E, Deniaud D, Adjali O. Mével M, et al. Mol Ther Methods Clin Dev. 2024 Jan 17;32(1):101187. doi: 10.1016/j.omtm.2024.101187. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38327809 Free PMC article.

48

Extracellular vesicle depletion and UGCG overexpression mitigate the cell density effect in HEK293 cell culture transfection.

Pérez-Rubio P, Lavado-García J, Bosch-Molist L, Romero EL, Cervera L, Gòdia F. Pérez-Rubio P, et al. Mol Ther Methods Clin Dev. 2024 Jan 17;32(1):101190. doi: 10.1016/j.omtm.2024.101190. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38327808 Free PMC article.

49

Expanded specific T cells to hypomutated regions of the SARS-CoV-2 using mRNA electroporated antigen-presenting cells.

Ogando-Rivas E, Castillo P, Yang C, Trivedi V, Zhang D, Pohl-Guimarães F, Liu R, Barpujari A, Candelario KM, Mendez-Gomez H, Sayour EJ, Mitchell DA. Ogando-Rivas E, et al. Mol Ther Methods Clin Dev. 2024 Jan 22;32(1):101192. doi: 10.1016/j.omtm.2024.101192. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38327807 Free PMC article.

50

Recombinant AAV genome size effect on viral vector production, purification, and thermostability.

Ibreljic N, Draper BE, Lawton CW. Ibreljic N, et al. Mol Ther Methods Clin Dev. 2024 Jan 17;32(1):101188. doi: 10.1016/j.omtm.2024.101188. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38327806 Free PMC article.

51

Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74.

Potter RA, Peterson EL, Griffin D, Cooper Olson G, Lewis S, Cochran K, Mendell JR, Rodino-Klapac LR. Potter RA, et al. Mol Ther Methods Clin Dev. 2024 Jan 23;32(1):101195. doi: 10.1016/j.omtm.2024.101195. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38327805 Free PMC article.

52

High-titer manufacturing of SARS-CoV-2 Spike-pseudotyped VSV in stirred-tank bioreactors.

Todesco HM, Gafuik C, John CM, Roberts EL, Borys BS, Pawluk A, Kallos MS, Potts KG, Mahoney DJ. Todesco HM, et al. Mol Ther Methods Clin Dev. 2024 Jan 17;32(1):101189. doi: 10.1016/j.omtm.2024.101189. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38327804 Free PMC article.

53

Manufacture of CD22 CAR T cells following positive versus negative selection results in distinct cytokine secretion profiles and γδ T cell output.

Song HW, Benzaoui M, Dwivedi A, Underwood S, Shao L, Achar S, Posarac V, Remley VA, Prochazkova M, Cai Y, Jin P, Somerville RP, Stroncek DF, Altan-Bonnet G, Shah NN, Chien CD, Taylor N, Highfill SL. Song HW, et al. Mol Ther Methods Clin Dev. 2023 Dec 11;32(1):101171. doi: 10.1016/j.omtm.2023.101171. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2023. PMID: 38298420 Free PMC article.

54

New perspectives for gene therapy of the X-linked form of Charcot-Marie-Tooth disease.

Caballé RB, Bortolozzi M. Caballé RB, et al. Mol Ther Methods Clin Dev. 2024 Jan 12;32(1):101184. doi: 10.1016/j.omtm.2023.101184. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38292668 Free PMC article. No abstract available.

55

Can mitochondria brown the lower-limb adipocytes?

Doulamis IP, Tzani A. Doulamis IP, et al. Mol Ther Methods Clin Dev. 2024 Jan 5;32(1):101181. doi: 10.1016/j.omtm.2023.101181. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38282897 Free PMC article. No abstract available.

56

Improving cell-specific recombination using AAV vectors in the murine CNS by capsid and expression cassette optimization.

Kawabata H, Konno A, Matsuzaki Y, Sato Y, Kawachi M, Aoki R, Tsutsumi S, Togai S, Kobayashi R, Horii T, Hatada I, Hirai H. Kawabata H, et al. Mol Ther Methods Clin Dev. 2024 Jan 10;32(1):101185. doi: 10.1016/j.omtm.2024.101185. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38282896 Free PMC article.

57

Efficient and sustained FOXP3 locus editing in hematopoietic stem cells as a therapeutic approach for IPEX syndrome.

Singh S, Pugliano CM, Honaker Y, Laird A, DeGottardi MQ, Lopez E, Lachkar S, Stoffers C, Sommer K, Khan IF, Rawlings DJ. Singh S, et al. Mol Ther Methods Clin Dev. 2023 Dec 26;32(1):101183. doi: 10.1016/j.omtm.2023.101183. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2023. PMID: 38282895 Free PMC article.

58

Long-term stability of clinical-grade lentiviral vectors for cell therapy.

Jadlowsky JK, Leskowitz R, McKenna S, Karar J, Ma Y, Dai A, Plesa G, Chen F, Alexander K, Petrella J, Gong N, Hwang WT, Farrelly O, Barber-Rotenberg J, Christensen S, Gonzalez VE, Chew A, Fraietta JA, June CH. Jadlowsky JK, et al. Mol Ther Methods Clin Dev. 2024 Jan 10;32(1):101186. doi: 10.1016/j.omtm.2024.101186. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38282894 Free PMC article.

59

The comparability tales: A phase-appropriate roadmap for CGT drug product development.

American Society of Gene and Cell Therapy (ASGCT) CMC Committee Members; Clément N, Kassim SH, Leblanc D, Spink K, Tomtishen J. American Society of Gene and Cell Therapy (ASGCT) CMC Committee Members, et al. Mol Ther Methods Clin Dev. 2023 Dec 5;32(1):101170. doi: 10.1016/j.omtm.2023.101170. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2023. PMID: 38268743 Free PMC article.

60

TAZing down metabolic mayhem: siRNAs against liver inflammation and fibrosis in humanized mice.

Weihs J, Rezvani M. Weihs J, et al. Mol Ther Methods Clin Dev. 2024 Jan 1;32(1):101182. doi: 10.1016/j.omtm.2023.101182. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38261959 Free PMC article. No abstract available.

61

Perspectives of the Friedreich ataxia community on gene therapy clinical trials.

Trantham SJ, Coker MA, Norman S, Crowley E, Berthy J, Byrne BJ, Subramony S, Lou X, Corti M. Trantham SJ, et al. Mol Ther Methods Clin Dev. 2023 Dec 18;32(1):101179. doi: 10.1016/j.omtm.2023.101179. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2023. PMID: 38261944 Free PMC article.

62

Genetic surgery for a cystic fibrosis-causing splicing mutation.

Bulcaen M, Carlon MS. Bulcaen M, et al. Mol Ther Methods Clin Dev. 2024 Jan 2;32(1):101177. doi: 10.1016/j.omtm.2023.101177. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38261927 Free PMC article. No abstract available.

63

Pulmonary macrophage transplant therapy in parenchymal lung diseases.

O'Callaghan M, McCarthy C. O'Callaghan M, et al. Mol Ther Methods Clin Dev. 2024 Jan 1;32(1):101180. doi: 10.1016/j.omtm.2023.101180. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38249935 Free PMC article. No abstract available.

64

AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models.

Murakami Y, Umeshita S, Imanishi K, Yoshioka Y, Ninomiya A, Sunabori T, Likhite S, Koike M, Meyer KC, Kinoshita T. Murakami Y, et al. Mol Ther Methods Clin Dev. 2023 Dec 14;32(1):101176. doi: 10.1016/j.omtm.2023.101176. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2023. PMID: 38225934 Free PMC article.

65

Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study.

Ryckman AE, Deschenes NM, Quinville BM, Osmon KJL, Mitchell M, Chen Z, Gray SJ, Walia JS. Ryckman AE, et al. Mol Ther Methods Clin Dev. 2023 Dec 5;32(1):101168. doi: 10.1016/j.omtm.2023.101168. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2023. PMID: 38205442 Free PMC article.

66

Lipid nanoparticle-encapsulated DNA vaccine robustly induce superior immune responses to the mRNA vaccine in Syrian hamsters.

Liao HC, Shen KY, Yang CH, Chiu FF, Chiang CY, Chai KM, Huang WC, Ho HM, Chen YH, Huang MS, Liao CL, Chen HW, Huang MH, Liu SJ. Liao HC, et al. Mol Ther Methods Clin Dev. 2023 Dec 5;32(1):101169. doi: 10.1016/j.omtm.2023.101169. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2023. PMID: 38187094 Free PMC article.

67

Nonviral delivery of nCas9 for "safe harbor" integration to treat MPS IVA.

Ou L. Ou L. Mol Ther Methods Clin Dev. 2023 Dec 29;32(1):101178. doi: 10.1016/j.omtm.2023.101178. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2023. PMID: 38178916 Free PMC article. No abstract available.

68

Regnase-1 overexpression as a therapeutic approach of Marfan syndrome.

Noormalal M, Schmiedel N, Bozoglu T, Matzen A, Hille S, Basha DI, Vijaya Shetty PM, Wolf A, Zaradzki M, Arif R, Pühler T, Lutter G, Wagner AH, Kupatt C, Frank D, Frey N, Remes A, Müller OJ. Noormalal M, et al. Mol Ther Methods Clin Dev. 2023 Dec 1;32(1):101163. doi: 10.1016/j.omtm.2023.101163. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2023. PMID: 38178915 Free PMC article.

69

Rescuing the cytolytic function of APDS1 patient T cells via TALEN-mediated PIK3CD gene correction.

Poggi L, Chentout L, Lizot S, Boyne A, Juillerat A, Moiani A, Luka M, Carbone F, Ménager M, Cavazzana M, Duchateau P, Valton J, Kracker S. Poggi L, et al. Mol Ther Methods Clin Dev. 2023 Oct 10;31:101133. doi: 10.1016/j.omtm.2023.101133. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38152700 Free PMC article.

70

Engineering a highly durable adeno-associated virus receptor for analytical applications.

Yoshida K, Tsunekawa Y, Kurihara K, Watanabe K, Makino-Manabe Y, Wada M, Tanaka T, Ide T, Okada T. Yoshida K, et al. Mol Ther Methods Clin Dev. 2023 Nov 14;31:101157. doi: 10.1016/j.omtm.2023.101157. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38152699 Free PMC article.

71

Inducible caspase 9-mediated suicide gene therapy using AAV6 vectors in a murine model of breast cancer.

Pathak S, Singh V, Kumar N, Jayandharan GR. Pathak S, et al. Mol Ther Methods Clin Dev. 2023 Nov 24;31:101166. doi: 10.1016/j.omtm.2023.101166. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38149057 Free PMC article.

72

Hepatocyte-targeted siTAZ therapy lowers liver fibrosis in NASH diet-fed chimeric mice with hepatocyte-humanized livers.

Wang X, Moore MP, Shi H, Miyata Y, Donnelly SK, Radiloff DR, Tabas I. Wang X, et al. Mol Ther Methods Clin Dev. 2023 Nov 23;31:101165. doi: 10.1016/j.omtm.2023.101165. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38144682 Free PMC article.

73

Iron oxide-coupled CRISPR-nCas9-based genome editing assessment in mucopolysaccharidosis IVA mice.

Leal AF, Celik B, Fnu N, Khan S, Tomatsu S, Alméciga-Díaz CJ. Leal AF, et al. Mol Ther Methods Clin Dev. 2023 Nov 7;31:101153. doi: 10.1016/j.omtm.2023.101153. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38107675 Free PMC article.

74

Genome length determination in adeno-associated virus vectors with mass photometry.

Hiemenz C, Baumeister N, Helbig C, Hawe A, Babutzka S, Michalakis S, Friess W, Menzen T. Hiemenz C, et al. Mol Ther Methods Clin Dev. 2023 Nov 19;31:101162. doi: 10.1016/j.omtm.2023.101162. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38094202 Free PMC article.

75

Erratum: Development of a gene edited next-generation hematopoietic cell transplant to enable acute myeloid leukemia treatment by solving off-tumor toxicity.

Lydeard JR, Lin MI, Ge HG, Halfond A, Wang S, Jones MB, Etchin J, Angelini G, Xavier-Ferrucio J, Lisle J, Salvadore K, Keschner Y, Mager H, Scherer J, Hu J, Mukherjee S, Chakraborty T. Lydeard JR, et al. Mol Ther Methods Clin Dev. 2023 Nov 30;31:101167. doi: 10.1016/j.omtm.2023.101167. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38094201 Free PMC article.

76

Analysis of vector genome integrations in multicentric lymphoma after AAV gene therapy in a severe hemophilia A dog.

Van Gorder L, Doshi BS, Willis E, Nichols TC, Cook E, Everett JK, Merricks EP, Arruda VR, Bushman FD, Callan MB, Samelson-Jones BJ. Van Gorder L, et al. Mol Ther Methods Clin Dev. 2023 Nov 14;31:101159. doi: 10.1016/j.omtm.2023.101159. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38094200 Free PMC article.

77

CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice.

Bortolussi G, Iaconcig A, Canarutto G, Porro F, Ferrucci F, Galletta C, Díaz-Muñoz C, Rawat V, De Caneva A, Olajide OJ, Zentilin L, Piazza S, Bočkor L, Muro AF. Bortolussi G, et al. Mol Ther Methods Clin Dev. 2023 Nov 19;31:101161. doi: 10.1016/j.omtm.2023.101161. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38094199 Free PMC article.

78

Predicted deleterious variants in the human genome relevant to gene therapy with adeno-associated virus vectors.

Rostami MR, Leopold PL, Vasquez JM, de Mulder Rougvie M, Al Shakaki A, Hssain AA, Robay A, Hackett NR, Mezey JG, Crystal RG. Rostami MR, et al. Mol Ther Methods Clin Dev. 2023 Oct 13;31:101136. doi: 10.1016/j.omtm.2023.101136. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38089635 Free PMC article.

79

Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications.

Roberts TC. Roberts TC. Mol Ther Methods Clin Dev. 2023 Nov 24;31:101160. doi: 10.1016/j.omtm.2023.101160. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38074414 Free PMC article. No abstract available.

80

Anti-tau intrabodies: From anti-tau immunoglobulins to the development of functional scFv intrabodies.

Rodrigues Martins D, Sha F, Van der Elst W, Shih PY, Devoght J, Van Kolen K, Mercken M, Van Broeck B, Declerck P, Theunis C. Rodrigues Martins D, et al. Mol Ther Methods Clin Dev. 2023 Nov 14;31:101158. doi: 10.1016/j.omtm.2023.101158. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38074413 Free PMC article.

81

Drug product attributes predict clinical efficacy in betibeglogene autotemcel gene therapy for β-thalassemia.

Whitney D, Shestopalov I, Fincker M, d'Anjou M, Kral K, Gayron M, Pierciey FJ, Colvin RA. Whitney D, et al. Mol Ther Methods Clin Dev. 2023 Nov 10;31:101155. doi: 10.1016/j.omtm.2023.101155. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38074412 Free PMC article.

82

Case report of selumetinib as a novel therapy in a neurofibromatosis type 2-associated ependymoma.

Blackwood N, Zetzmann C, Trevino CR. Blackwood N, et al. Mol Ther Methods Clin Dev. 2023 Nov 10;31:101156. doi: 10.1016/j.omtm.2023.101156. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38058737 Free PMC article.

83

Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy.

Shoti J, Qing K, Keeler GD, Duan D, Byrne BJ, Srivastava A. Shoti J, et al. Mol Ther Methods Clin Dev. 2023 Nov 2;31:101147. doi: 10.1016/j.omtm.2023.101147. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38046199 Free PMC article.

84

Fast HPLC-based affinity method to determine capsid titer and full/empty ratio of adeno-associated viral vectors.

Heckel J, Martinez A, Elger C, Haindl M, Leiss M, Ruppert R, Williams C, Hubbuch J, Graf T. Heckel J, et al. Mol Ther Methods Clin Dev. 2023 Nov 2;31:101148. doi: 10.1016/j.omtm.2023.101148. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38046198 Free PMC article.

85

Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II.

Catalano F, Vlaar EC, Katsavelis D, Dammou Z, Huizer TF, van den Bosch JC, Hoogeveen-Westerveld M, van den Hout HJMP, Oussoren E, Ruijter GJG, Schaaf G, Pike-Overzet K, Staal FJT, van der Ploeg AT, Pijnappel WWMP. Catalano F, et al. Mol Ther Methods Clin Dev. 2023 Nov 2;31:101149. doi: 10.1016/j.omtm.2023.101149. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38033460 Free PMC article.

86

Brain-targeted ex vivo lentiviral gene therapy: Implications for MPS and beyond.

Bradbury AM. Bradbury AM. Mol Ther Methods Clin Dev. 2023 Oct 28;31:101137. doi: 10.1016/j.omtm.2023.101137. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027070 Free PMC article. No abstract available.

87

Bringing base editing to the clinic: The next generation of genome editors.

Flugel CL, Abou-El-Enein M. Flugel CL, et al. Mol Ther Methods Clin Dev. 2023 Nov 3;31:101138. doi: 10.1016/j.omtm.2023.101138. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027069 Free PMC article. No abstract available.

88

Generation of hepatitis C virus-resistant liver cells by genome editing-mediated stable expression of RNA aptamer.

Kim TH, Lee SW. Kim TH, et al. Mol Ther Methods Clin Dev. 2023 Nov 7;31:101151. doi: 10.1016/j.omtm.2023.101151. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027068 Free PMC article.

89

CRISPR-Cas9-based non-viral gene editing therapy for topical treatment of recessive dystrophic epidermolysis bullosa.

Wang X, Wang X, Li Y, A S, Qiu B, Bushmalyova A, He Z, Wang W, Lara-Sáez I. Wang X, et al. Mol Ther Methods Clin Dev. 2023 Oct 13;31:101134. doi: 10.1016/j.omtm.2023.101134. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027067 Free PMC article.

90

Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and nonhuman primates.

Zhang L, Wang H, Xun M, Tang H, Wang J, Lv J, Zhu B, Chen Y, Wang D, Hu S, Gao Z, Liu J, Chen ZY, Chen B, Li H, Shu Y. Zhang L, et al. Mol Ther Methods Clin Dev. 2023 Nov 10;31:101154. doi: 10.1016/j.omtm.2023.101154. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027066 Free PMC article.

91

AAV2-antiVEGFscFv gene therapy for retinal neovascularization.

Han N, Xu X, Liu Y, Luo G. Han N, et al. Mol Ther Methods Clin Dev. 2023 Oct 29;31:101145. doi: 10.1016/j.omtm.2023.101145. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027065 Free PMC article.

92

Development of a gene edited next-generation hematopoietic cell transplant to enable acute myeloid leukemia treatment by solving off-tumor toxicity.

Lydeard JR, Lin MI, Ge HG, Halfond A, Wang S, Jones MB, Etchin J, Angelini G, Xavier-Ferrucio J, Lisle J, Salvadore K, Keschner Y, Mager H, Scherer J, Hu J, Mukherjee S, Chakraborty T. Lydeard JR, et al. Mol Ther Methods Clin Dev. 2023 Oct 13;31:101135. doi: 10.1016/j.omtm.2023.101135. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027064 Free PMC article.

93

A C1qTNF3 collagen domain fusion chaperones diverse secreted proteins and anti-Aβ scFvs: Applications for gene therapies.

Moore BD, Ran Y, Goodwin MS, Komatineni K, McFarland KN, Dillon K, Charles C, Ryu D, Liu X, Prokop S, Giasson BI, Golde TE, Levites Y. Moore BD, et al. Mol Ther Methods Clin Dev. 2023 Oct 31;31:101146. doi: 10.1016/j.omtm.2023.101146. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027063 Free PMC article.

94

New dawn of cellular therapies in autoimmune diseases.

Wagner DL, Ostendorf L. Wagner DL, et al. Mol Ther Methods Clin Dev. 2023 Nov 8;31:101141. doi: 10.1016/j.omtm.2023.101141. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027062 Free PMC article. No abstract available.

95

Mitochondrial transplantation reduces lower limb ischemia-reperfusion injury by increasing skeletal muscle energy and adipocyte browning.

Zeng J, Liu J, Ni H, Zhang L, Wang J, Li Y, Jiang W, Wu Z, Zhou M. Zeng J, et al. Mol Ther Methods Clin Dev. 2023 Nov 7;31:101152. doi: 10.1016/j.omtm.2023.101152. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027061 Free PMC article.

96

Molecular and functional correction of a deep intronic splicing mutation in CFTR by CRISPR-Cas9 gene editing.

Walker AJ, Graham C, Greenwood M, Woodall M, Maeshima R, O'Hara-Wright M, Sanz DJ, Guerrini I, Aldossary AM, O'Callaghan C, Baines DL, Harrison PT, Hart SL. Walker AJ, et al. Mol Ther Methods Clin Dev. 2023 Oct 18;31:101140. doi: 10.1016/j.omtm.2023.101140. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027060 Free PMC article.

97

A novel FOXP3 knockout-humanized mouse model for pre-clinical safety and efficacy evaluation of Treg-like cell products.

Sato Y, Nathan A, Shipp S, Wright JF, Tate KM, Wani P, Roncarolo MG, Bacchetta R. Sato Y, et al. Mol Ther Methods Clin Dev. 2023 Nov 7;31:101150. doi: 10.1016/j.omtm.2023.101150. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027059 Free PMC article.

98

Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model.

Gushchina LV, Bradley AJ, Vetter TA, Lay JW, Rohan NL, Frair EC, Wein N, Flanigan KM. Gushchina LV, et al. Mol Ther Methods Clin Dev. 2023 Oct 26;31:101144. doi: 10.1016/j.omtm.2023.101144. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027058 Free PMC article.

99

Multiparametric domain insertional profiling of adeno-associated virus VP1.

Hoffmann MD, Zdechlik AC, He Y, Nedrud D, Aslanidi G, Gordon W, Schmidt D. Hoffmann MD, et al. Mol Ther Methods Clin Dev. 2023 Oct 26;31:101143. doi: 10.1016/j.omtm.2023.101143. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027057 Free PMC article.

100

Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering.

Kitte R, Rabel M, Geczy R, Park S, Fricke S, Koehl U, Tretbar US. Kitte R, et al. Mol Ther Methods Clin Dev. 2023 Oct 18;31:101139. doi: 10.1016/j.omtm.2023.101139. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027056 Free PMC article.

101

Enhancement of recombinant adeno-associated virus activity by improved stoichiometry and homogeneity of capsid protein assembly.

Onishi T, Nonaka M, Maruno T, Yamaguchi Y, Fukuhara M, Torisu T, Maeda M, Abbatiello S, Haris A, Richardson K, Giles K, Preece S, Yamano-Adachi N, Omasa T, Uchiyama S. Onishi T, et al. Mol Ther Methods Clin Dev. 2023 Oct 24;31:101142. doi: 10.1016/j.omtm.2023.101142. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027055 Free PMC article.

102

PCR-based analytics of gene therapies using adeno-associated virus vectors: Considerations for cGMP method development.

Blay E, Hardyman E, Morovic W. Blay E, et al. Mol Ther Methods Clin Dev. 2023 Oct 27;31:101132. doi: 10.1016/j.omtm.2023.101132. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37964893 Free PMC article. Review.

103

A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies.

Pan Y, Rohde M, Zeitler J, Namburi SVS, Cao L, Hu J, Meyer K, Lu Y. Pan Y, et al. Mol Ther Methods Clin Dev. 2023 Oct 10;31:101126. doi: 10.1016/j.omtm.2023.101126. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37920239 Free PMC article.

104

Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model.

Benatti HR, Prestigiacomo RD, Taghian T, Miller R, King R, Gounis MJ, Celik U, Bertrand S, Tuominen S, Bierfeldt L, Parsley E, Gallagher J, Hall EF, McElroy AW, Sena-Esteves M, Khvorova A, Aronin N, Gray-Edwards HL. Benatti HR, et al. Mol Ther Methods Clin Dev. 2023 Sep 26;31:101122. doi: 10.1016/j.omtm.2023.101122. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37920238 Free PMC article.

105

Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy.

Ellison S, Liao A, Gleitz HFE, Parker H, Booth L, Robinson J, Wood S, Taylor J, Holley R, Bigger BW. Ellison S, et al. Mol Ther Methods Clin Dev. 2023 Oct 20;31:101127. doi: 10.1016/j.omtm.2023.101127. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37920237 Free PMC article.

106

Prostaglandin E2 as transduction enhancer affects competitive engraftment of human hematopoietic stem and progenitor cells.

Poletti V, Montepeloso A, Pellin D, Biffi A. Poletti V, et al. Mol Ther Methods Clin Dev. 2023 Oct 11;31:101131. doi: 10.1016/j.omtm.2023.101131. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37920236 Free PMC article.

107

Large-scale manufacturing of base-edited chimeric antigen receptor T cells.

Woodruff R, Parekh F, Lamb K, Mekkaoui L, Allen C, Smetanova K, Huang J, Williams A, Toledo GS, Lilova K, Roddie C, Sillibourne J, Pule M. Woodruff R, et al. Mol Ther Methods Clin Dev. 2023 Oct 5;31:101123. doi: 10.1016/j.omtm.2023.101123. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37886606 Free PMC article.

108

The hunt for novel AAV capsids with improved cardiac tropism.

Miguel-Dos-Santos R, Cingolani E. Miguel-Dos-Santos R, et al. Mol Ther Methods Clin Dev. 2023 Oct 20;31:101124. doi: 10.1016/j.omtm.2023.101124. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37886605 Free PMC article. No abstract available.

109

Characterization and AAV-mediated CRB gene augmentation in human-derived CRB1^KO and CRB1^KOCRB2^+/- retinal organoids.

Boon N, Lu X, Andriessen CA, Orlovà M, Quinn PMJ, Boon CJF, Wijnholds J. Boon N, et al. Mol Ther Methods Clin Dev. 2023 Oct 10;31:101128. doi: 10.1016/j.omtm.2023.101128. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37886604 Free PMC article.

110

Clinical holds for cell and gene therapy trials: Risks, impact, and lessons learned.

Wills CA, Drago D, Pietrusko RG. Wills CA, et al. Mol Ther Methods Clin Dev. 2023 Oct 20;31:101125. doi: 10.1016/j.omtm.2023.101125. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37886603 Free PMC article. Review.

111

Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice.

Chang H, Du A, Jiang J, Ren L, Liu N, Zhou X, Liang J, Gao G, Wang D. Chang H, et al. Mol Ther Methods Clin Dev. 2023 Oct 7;31:101129. doi: 10.1016/j.omtm.2023.101129. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37886602 Free PMC article.

112

Adeno-associated viral capsid stability on anion exchange chromatography column and its impact on empty and full capsid separation.

Khanal O, Kumar V, Jin M. Khanal O, et al. Mol Ther Methods Clin Dev. 2023 Sep 19;31:101112. doi: 10.1016/j.omtm.2023.101112. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37868210 Free PMC article.

113

Efficient long-term multilineage engraftment of CD33-edited hematopoietic stem/progenitor cells in nonhuman primates.

Petty NE, Radtke S, Fields E, Humbert O, Llewellyn MJ, Laszlo GS, Zhu H, Jerome KR, Walter RB, Kiem HP. Petty NE, et al. Mol Ther Methods Clin Dev. 2023 Sep 26;31:101121. doi: 10.1016/j.omtm.2023.101121. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37868209 Free PMC article.

114

Erratum: Immortalized human myoblast cell lines for the delivery of therapeutic proteins using encapsulated cell technology.

Lathuiliere A, Vernet R, Charrier E, Urwyler M, Von Rohr O, Belkouch MC, Saingier V, Bouvarel T, Guillarme D, Engel A, Salmon P, Laumonier T, Grogg J, Mach N. Lathuiliere A, et al. Mol Ther Methods Clin Dev. 2023 Oct 12;31:101130. doi: 10.1016/j.omtm.2023.101130. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37868208 Free PMC article.

115

Rationale for using centralized transduction inhibition assays in three phase 3 rAAV gene therapy clinical trials.

Schulz M, Bashirians G, Cheng SH, Levy DI, Lundie M, Wilcox L, Winburn I, Somanathan S. Schulz M, et al. Mol Ther Methods Clin Dev. 2023 Oct 11;31:101119. doi: 10.1016/j.omtm.2023.101119. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37868207 Free PMC article. No abstract available.

116

Optogenetic targeting of AII amacrine cells restores retinal computations performed by the inner retina.

Khabou H, Orendorff E, Trapani F, Rucli M, Desrosiers M, Yger P, Dalkara D, Marre O. Khabou H, et al. Mol Ther Methods Clin Dev. 2023 Sep 13;31:101107. doi: 10.1016/j.omtm.2023.09.003. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37868206 Free PMC article.

117

Inhibition of DNA-dependent protein kinase catalytic subunit boosts rAAV transduction of polarized human airway epithelium.

Ning K, Zhang X, Feng Z, Hao S, Kuz CA, Cheng F, Park SY, McFarlin S, Engelhardt JF, Yan Z, Qiu J. Ning K, et al. Mol Ther Methods Clin Dev. 2023 Sep 21;31:101115. doi: 10.1016/j.omtm.2023.101115. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37841417 Free PMC article.

118

Universal ddPCR-based assay for the determination of lentivirus infectious titer and lenti-modified cell vector copy number.

Kandell J, Milian S, Snyder R, Lakshmipathy U. Kandell J, et al. Mol Ther Methods Clin Dev. 2023 Sep 23;31:101120. doi: 10.1016/j.omtm.2023.101120. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37841416 Free PMC article.

119

Mini-TCRs: Truncated T cell receptors to generate T cells from induced pluripotent stem cells.

Takayanagi SI, Wang B, Hasegawa S, Nishikawa S, Fukumoto K, Nakano K, Chuganji S, Kato Y, Kamibayashi S, Minagawa A, Kunisato A, Nozawa H, Kaneko S. Takayanagi SI, et al. Mol Ther Methods Clin Dev. 2023 Sep 16;31:101109. doi: 10.1016/j.omtm.2023.101109. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37822720 Free PMC article.

120

A T cell-targeted multi-antigen vaccine generates robust cellular and humoral immunity against SARS-CoV-2 infection.

Boulton S, Poutou J, Gill R, Alluqmani N, He X, Singaravelu R, Crupi MJF, Petryk J, Austin B, Angka L, Taha Z, Teo I, Singh S, Jamil R, Marius R, Martin N, Jamieson T, Azad T, Diallo JS, Ilkow CS, Bell JC. Boulton S, et al. Mol Ther Methods Clin Dev. 2023 Sep 16;31:101110. doi: 10.1016/j.omtm.2023.101110. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37822719 Free PMC article.

121

Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy.

Day JW, Mendell JR, Burghes AHM, van Olden RW, Adhikary RR, Dilly KW. Day JW, et al. Mol Ther Methods Clin Dev. 2023 Sep 20;31:101117. doi: 10.1016/j.omtm.2023.101117. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37822718 Free PMC article.

122

Unveiling the secrets of adeno-associated virus: novel high-throughput approaches for the quantification of multiple serotypes.

Meierrieks F, Kour A, Pätz M, Pflanz K, Wolff MW, Pickl A. Meierrieks F, et al. Mol Ther Methods Clin Dev. 2023 Sep 20;31:101118. doi: 10.1016/j.omtm.2023.101118. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37822717 Free PMC article.

123

Archival skin biopsy specimens as a tool for miRNA-based diagnosis: Technical and post-analytical considerations.

Andelic M, Marchi M, Marcuzzo S, Lombardi R, Faber CG, Lauria G, Salvi E. Andelic M, et al. Mol Ther Methods Clin Dev. 2023 Sep 16;31:101116. doi: 10.1016/j.omtm.2023.101116. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37808256 Free PMC article.

124

Production of therapeutic levels of human FIX-R338L by engineered B cells using GMP-compatible medium.

David M, Monteferrario D, Saviane G, Jeanneau C, Marchetti I, Dupont CF, Dumont C, Fontenot JD, Rosa M, Fenard D. David M, et al. Mol Ther Methods Clin Dev. 2023 Sep 19;31:101111. doi: 10.1016/j.omtm.2023.101111. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37790246 Free PMC article.

125

Development of an automated manufacturing process for large-scale production of autologous T cell therapies.

Francis N, Braun M, Neagle S, Peiffer S, Bohn A, Rosenthal A, Olbrich T, Lollies S, Ilsmann K, Hauck C, Gerstmayer B, Weber S, Kirkpatrick A. Francis N, et al. Mol Ther Methods Clin Dev. 2023 Sep 16;31:101114. doi: 10.1016/j.omtm.2023.101114. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37790245 Free PMC article.

126

Identification of a small molecule for enhancing lentiviral transduction of T cells.

Malach P, Kay C, Tinworth C, Patel F, Joosse B, Wade J, Rosa do Carmo M, Donovan B, Brugman M, Montiel-Equihua C, Francis N. Malach P, et al. Mol Ther Methods Clin Dev. 2023 Sep 19;31:101113. doi: 10.1016/j.omtm.2023.101113. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37790244 Free PMC article.

127

iMSC: One leap closer to clinical applications.

Hoang DM. Hoang DM. Mol Ther Methods Clin Dev. 2023 Sep 22;31:101095. doi: 10.1016/j.omtm.2023.08.014. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37766792 Free PMC article. No abstract available.

128

AAV-BDNF gene therapy ameliorates a hypothalamic neuroinflammatory signature in the Magel2-null model of Prader-Willi syndrome.

Queen NJ, Huang W, Zou X, Mo X, Cao L. Queen NJ, et al. Mol Ther Methods Clin Dev. 2023 Sep 13;31:101108. doi: 10.1016/j.omtm.2023.09.004. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37766791 Free PMC article.

129

Oxidative stress induced by sustained supraphysiological intrastriatal GDNF delivery is prevented by dose regulation.

Azevedo MD, Prince N, Humbert-Claude M, Mesa-Infante V, Jeanneret C, Golzne V, De Matos K, Jamot BB, Magara F, Gonzalez-Hernandez T, Tenenbaum L. Azevedo MD, et al. Mol Ther Methods Clin Dev. 2023 Sep 9;31:101106. doi: 10.1016/j.omtm.2023.09.002. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37766790 Free PMC article.

130

Breaching the blood-brain barrier: AAV triggers dose-dependent toxicity in the brain.

Stone D, Aubert M, Jerome KR. Stone D, et al. Mol Ther Methods Clin Dev. 2023 Sep 20;31:101105. doi: 10.1016/j.omtm.2023.09.001. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37753219 Free PMC article. No abstract available.

131

High-titer AAV disrupts cerebrovascular integrity and induces lymphocyte infiltration in adult mouse brain.

Guo Y, Chen J, Ji W, Xu L, Xie Y, He S, Lai C, Hou K, Li Z, Chen G, Wu Z. Guo Y, et al. Mol Ther Methods Clin Dev. 2023 Aug 28;31:101102. doi: 10.1016/j.omtm.2023.08.021. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37753218 Free PMC article.

132

Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9.

Lisjak M, Iaconcig A, Guarnaccia C, Vicidomini A, Moretti L, Collaud F, Ronzitti G, Zentilin L, Muro AF. Lisjak M, et al. Mol Ther Methods Clin Dev. 2023 Aug 28;31:101103. doi: 10.1016/j.omtm.2023.08.022. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37744006 Free PMC article.

133

Cytokine and reactivity profiles in SLE patients following anti-CD19 CART therapy.

Nunez D, Patel D, Volkov J, Wong S, Vorndran Z, Müller F, Aigner M, Völkl S, Mackensen A, Schett G, Basu S. Nunez D, et al. Mol Ther Methods Clin Dev. 2023 Sep 1;31:101104. doi: 10.1016/j.omtm.2023.08.023. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37744005 Free PMC article.

134

Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia.

Chandler RJ, Di Pasquale G, Choi EY, Chang D, Smith SN, Sloan JL, Hoffmann V, Li L, Chiorini JA, Venditti CP. Chandler RJ, et al. Mol Ther Methods Clin Dev. 2023 Jun 25;30:181-190. doi: 10.1016/j.omtm.2023.06.008. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37746248 Free PMC article.

135

Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy.

Le Guiner C, Xiao X, Larcher T, Lafoux A, Huchet C, Toumaniantz G, Adjali O, Anegon I, Remy S, Grieger J, Li J, Farrokhi V, Neubert H, Owens J, McIntyre M, Moullier P, Samulski RJ. Le Guiner C, et al. Mol Ther Methods Clin Dev. 2023 May 18;30:30-47. doi: 10.1016/j.omtm.2023.05.017. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37746247 Free PMC article.

136

Correlating physicochemical and biological properties to define critical quality attributes of a rAAV vaccine candidate.

Kumar P, Wang M, Kumru OS, Hickey JM, Sanmiguel J, Zabaleta N, Vandenberghe LH, Joshi SB, Volkin DB. Kumar P, et al. Mol Ther Methods Clin Dev. 2023 Jun 12;30:103-121. doi: 10.1016/j.omtm.2023.06.004. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37746246 Free PMC article.

137

Mechanistic modeling explains the production dynamics of recombinant adeno-associated virus with the baculovirus expression vector system.

Destro F, Joseph J, Srinivasan P, Kanter JM, Neufeld C, Wolfrum JM, Barone PW, Springs SL, Sinskey AJ, Cecchini S, Kotin RM, Braatz RD. Destro F, et al. Mol Ther Methods Clin Dev. 2023 Jun 2;30:122-146. doi: 10.1016/j.omtm.2023.05.019. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37746245 Free PMC article.

138

In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans.

Schwartz MK, Likhite S, Vetter TA, Baird MC, McGovern V, Sierra Delgado A, Mendel T, Burghes A, Meyer KC. Schwartz MK, et al. Mol Ther Methods Clin Dev. 2023 May 16;30:16-29. doi: 10.1016/j.omtm.2023.05.016. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37746244 Free PMC article.

139

Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy.

Emami MR, Espinoza A, Young CS, Ma F, Farahat PK, Felgner PL, Chamberlain JS, Xu X, Pyle AD, Pellegrini M, Villalta SA, Spencer MJ. Emami MR, et al. Mol Ther Methods Clin Dev. 2023 Jun 12;30:90-102. doi: 10.1016/j.omtm.2023.06.002. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37746243 Free PMC article.

140

CRISPR-Cas9 homology-independent targeted integration of exons 1-19 restores full-length dystrophin in mice.

Stephenson AA, Nicolau S, Vetter TA, Dufresne GP, Frair EC, Sarff JE, Wheeler GL, Kelly BJ, White P, Flanigan KM. Stephenson AA, et al. Mol Ther Methods Clin Dev. 2023 Aug 18;30:486-499. doi: 10.1016/j.omtm.2023.08.009. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37706184 Free PMC article.

141

Expression of gain-of-function CFTR in cystic fibrosis airway cells restores epithelial function better than wild-type or codon-optimized CFTR.

Woodall M, Tarran R, Lee R, Anfishi H, Prins S, Counsell J, Vergani P, Hart S, Baines D. Woodall M, et al. Mol Ther Methods Clin Dev. 2023 Aug 12;30:593-605. doi: 10.1016/j.omtm.2023.08.006. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37701179 Free PMC article.

142

Development of an in vitro genotoxicity assay to detect retroviral vector-induced lymphoid insertional mutants.

Bastone AL, Dziadek V, John-Neek P, Mansel F, Fleischauer J, Agyeman-Duah E, Schaudien D, Dittrich-Breiholz O, Schwarzer A, Schambach A, Rothe M. Bastone AL, et al. Mol Ther Methods Clin Dev. 2023 Aug 22;30:515-533. doi: 10.1016/j.omtm.2023.08.017. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37693949 Free PMC article.

143

Efficient gene transduction in pigs and macaques with the engineered AAV vector AAV.GT5 for hemophilia B gene therapy.

Kashiwakura Y, Endo K, Ugajin A, Kikuchi T, Hishikawa S, Nakamura H, Katakai Y, Baatartsogt N, Hiramoto T, Hayakawa M, Kamoshita N, Yamazaki S, Kume A, Mori H, Sata N, Sakata Y, Muramatsu SI, Ohmori T. Kashiwakura Y, et al. Mol Ther Methods Clin Dev. 2023 Aug 22;30:502-514. doi: 10.1016/j.omtm.2023.08.016. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37693948 Free PMC article.

144

Human retina-in-a-dish: Unlocking the potential to study mechanisms of inherited retinal disease.

Yee T, Wert KJ. Yee T, et al. Mol Ther Methods Clin Dev. 2023 Sep 3;30:573-575. doi: 10.1016/j.omtm.2023.08.019. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37693947 Free PMC article. No abstract available.

145

Dual-AAV vector-mediated expression of MYO7A improves vestibular function in a mouse model of Usher syndrome 1B.

Lau SC, Grati M, Isgrig K, Sinan M, Calabro KR, Zhu J, Ishibashi Y, Ozgur Z, Wafa T, Belyantseva IA, Fitzgerald T, Friedman TB, Boye SL, Boye SE, Chien WW. Lau SC, et al. Mol Ther Methods Clin Dev. 2023 Aug 21;30:534-545. doi: 10.1016/j.omtm.2023.08.012. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37693946 Free PMC article.

146

CRISPR-Cas9-mediated exon skipping as a cardioprotective strategy in Duchenne muscular dystrophy.

Wilton-Clark H, Yokota T. Wilton-Clark H, et al. Mol Ther Methods Clin Dev. 2023 Sep 4;30:500-501. doi: 10.1016/j.omtm.2023.08.013. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37693945 Free PMC article. No abstract available.

147

Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo.

Asperti C, Canarutto D, Porcellini S, Sanvito F, Cecere F, Vavassori V, Ferrari S, Rovelli E, Albano L, Jacob A, Sergi Sergi L, Montaldo E, Ferrua F, González-Granado LI, Lougaris V, Badolato R, Finocchi A, Villa A, Radrizzani M, Naldini L. Asperti C, et al. Mol Ther Methods Clin Dev. 2023 Aug 23;30:546-557. doi: 10.1016/j.omtm.2023.08.020. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37693944 Free PMC article.

148

Modifying immune responses to adeno-associated virus vectors by capsid engineering.

Bentler M, Hardet R, Ertelt M, Rudolf D, Kaniowska D, Schneider A, Vondran FWR, Schoeder CT, Delphin M, Lucifora J, Ott M, Hacker UT, Adriouch S, Büning H. Bentler M, et al. Mol Ther Methods Clin Dev. 2023 Aug 22;30:576-592. doi: 10.1016/j.omtm.2023.08.015. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37693943 Free PMC article.

149

Apolipoprotein A-I vascular gene therapy reduces vein-graft atherosclerosis.

Bi L, Wacker BK, Komandur K, Sanford N, Dichek DA. Bi L, et al. Mol Ther Methods Clin Dev. 2023 Aug 22;30:558-572. doi: 10.1016/j.omtm.2023.08.018. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37693942 Free PMC article.

150

Slow and steady wins the race: Optimizing lentiviral vector production for efficient clinical-scale manufacturing.

McCarron A. McCarron A. Mol Ther Methods Clin Dev. 2023 Aug 29;30:484-485. doi: 10.1016/j.omtm.2023.08.011. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37681060 Free PMC article. No abstract available.

151

Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec.

Asher D, Dai D, Klimchak AC, Sedita LE, Gooch KL, Rodino-Klapac L. Asher D, et al. Mol Ther Methods Clin Dev. 2023 Aug 9;30:474-483. doi: 10.1016/j.omtm.2023.08.002. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37674905 Free PMC article. Review.

152

Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes.

Kok CY, Tsurusaki S, Cabanes-Creus M, Igoor S, Rao R, Skelton R, Liao SHY, Ginn SL, Knight M, Scott S, Mietzsch M, Fitzsimmons R, Miller J, Mohamed TMA, McKenna R, Chong JJH, Hill AP, Hudson JE, Alexander IE, Lisowski L, Kizana E. Kok CY, et al. Mol Ther Methods Clin Dev. 2023 Aug 18;30:459-473. doi: 10.1016/j.omtm.2023.08.010. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37674904 Free PMC article.

153

Toward lentiviral vectors for antiangiogenic ocular gene therapy.

Askou AL, Jakobsen TS, Corydon TJ. Askou AL, et al. Mol Ther Methods Clin Dev. 2023 Aug 26;30:443-446. doi: 10.1016/j.omtm.2023.08.007. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37663649 Free PMC article. No abstract available.

154

Necessity of strengthening the current clinical regulatory for companion diagnostics: An institutional comparison of the FDA, EMA, and MFDS.

Kang SL, Woo JH, Kim NH, Kwon JY, Kim SM. Kang SL, et al. Mol Ther Methods Clin Dev. 2023 Aug 16;30:447-458. doi: 10.1016/j.omtm.2023.08.008. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37663648 Free PMC article. Review.

155

Toward effective hematopoietic stem cell gene therapies: Optimized conditioning regimen and stem cell source in harmony.

Demirci S, Tisdale JF. Demirci S, et al. Mol Ther Methods Clin Dev. 2023 Aug 22;30:411-412. doi: 10.1016/j.omtm.2023.08.005. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37663647 Free PMC article. No abstract available.

156

Establishment of UGT1A1-knockout human iPS-derived hepatic organoids for UGT1A1-specific kinetics and toxicity evaluation.

Shintani T, Imamura C, Ueyama-Toba Y, Inui J, Watanabe A, Mizuguchi H. Shintani T, et al. Mol Ther Methods Clin Dev. 2023 Aug 11;30:429-442. doi: 10.1016/j.omtm.2023.08.003. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37663646 Free PMC article.

157

Selection of viral capsids and promoters affects the efficacy of rescue of Tmprss3-deficient cochlea.

Aaron KA, Pekrun K, Atkinson PJ, Billings SE, Abitbol JM, Lee IA, Eltawil Y, Chen YS, Dong W, Nelson RF, Kay MA, Cheng AG. Aaron KA, et al. Mol Ther Methods Clin Dev. 2023 Aug 11;30:413-428. doi: 10.1016/j.omtm.2023.08.004. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37663645 Free PMC article.

158

Gene replacement therapy in two Golgi-retained CMT1X mutants before and after the onset of demyelinating neuropathy.

Kagiava A, Karaiskos C, Lapathitis G, Heslegrave A, Sargiannidou I, Zetterberg H, Bosch A, Kleopa KA. Kagiava A, et al. Mol Ther Methods Clin Dev. 2023 Aug 2;30:377-393. doi: 10.1016/j.omtm.2023.07.011. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37645436 Free PMC article.

159

Crossing the gates of Babylon: Brain-penetrating enzyme replacement for lysosomal disorders.

Baldo G. Baldo G. Mol Ther Methods Clin Dev. 2023 Aug 10;30:315-316. doi: 10.1016/j.omtm.2023.07.008. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37637386 Free PMC article. No abstract available.

160

iPSC-derived mesenchymal stem cells attenuate cerebral ischemia-reperfusion injury by inhibiting inflammatory signaling and oxidative stress.

Arakawa M, Sakamoto Y, Miyagawa Y, Nito C, Takahashi S, Nitahara-Kasahara Y, Suda S, Yamazaki Y, Sakai M, Kimura K, Okada T. Arakawa M, et al. Mol Ther Methods Clin Dev. 2023 Jul 15;30:333-349. doi: 10.1016/j.omtm.2023.07.005. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37637385 Free PMC article.

161

Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease.

Xu L, Lahiri P, Skowronski J, Bhatia N, Lattanzi A, Porteus MH. Xu L, et al. Mol Ther Methods Clin Dev. 2023 Jul 27;30:317-331. doi: 10.1016/j.omtm.2023.07.009. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37637384 Free PMC article.

162

Erratum: AAV vector production: Troublesome host innate responses in another setting.

Wright JF. Wright JF. Mol Ther Methods Clin Dev. 2023 Aug 11;30:332. doi: 10.1016/j.omtm.2023.08.001. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37637383 Free PMC article.

163

Combined clarification and affinity capture using magnetic resin enables efficient separation of rAAV5 from cell lysate.

Turco F, Wegelius A, Lind O, Norrman N, Magnusson AC, Sund-Lundström C, Norén B, Hedberg J, Palmgren R. Turco F, et al. Mol Ther Methods Clin Dev. 2023 Jul 28;30:394-402. doi: 10.1016/j.omtm.2023.07.010. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37637382 Free PMC article.

164

Fully closed cell sorter for immune cell therapy manufacturing.

Matsumoto M, Tashiro S, Ito T, Takahashi K, Hashimoto G, Kajihara J, Miyahara Y, Shiku H, Katsumoto Y. Matsumoto M, et al. Mol Ther Methods Clin Dev. 2023 Aug 2;30:367-376. doi: 10.1016/j.omtm.2023.07.012. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37637381 Free PMC article.

165

Lentiviral delivered aflibercept OXB-203 for treatment of neovascular AMD.

Iqball S, Beck DK, Devarajan G, Khoo CP, O'Connor DM, Ellis S, Guzman E, Mitrophanous KA, Lad Y. Iqball S, et al. Mol Ther Methods Clin Dev. 2023 Jul 15;30:350-366. doi: 10.1016/j.omtm.2023.07.001. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37637380 Free PMC article.

166

Detection and quantification of integrated vector copy number by multiplex droplet digital PCR in dual-transduced CAR T cells.

Wang W, Al-Hajj M, Alavi AS. Wang W, et al. Mol Ther Methods Clin Dev. 2023 Jul 15;30:403-410. doi: 10.1016/j.omtm.2023.07.003. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37622159 Free PMC article.

167

Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation.

Corti M, Byrne BJ, Gessler DJ, Thompson G, Norman S, Lammers J, Coleman KE, Liberati C, Elder ME, Escolar ML, Tuna IS, Mesaros C, Kleiner GI, Barbouth DS, Gray-Edwards HL, Clement N, Cleaver BD, Gao G. Corti M, et al. Mol Ther Methods Clin Dev. 2023 Jun 19;30:303-314. doi: 10.1016/j.omtm.2023.06.001. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37601414 Free PMC article.

168

Partial genome content within rAAVs impacts performance in a cell assay-dependent manner.

Troxell B, Jaslow SL, Tsai IW, Sullivan C, Draper BE, Jarrold MF, Lindsey K, Blue L. Troxell B, et al. Mol Ther Methods Clin Dev. 2023 Jul 19;30:288-302. doi: 10.1016/j.omtm.2023.07.007. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37583716 Free PMC article.

169

Efficient manufacturing and engraftment of CCR5 gene-edited HSPCs following busulfan conditioning in nonhuman primates.

Murray J, Einhaus T, Venkataraman R, Radtke S, Zhen A, Carrillo MA, Kitchen SG, Peterson CW, Kiem HP. Murray J, et al. Mol Ther Methods Clin Dev. 2023 Jul 18;30:276-287. doi: 10.1016/j.omtm.2023.07.006. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37575091 Free PMC article.

170

Lipid nanoparticle mRNA systems containing high levels of sphingomyelin engender higher protein expression in hepatic and extra-hepatic tissues.

Chander N, Basha G, Yan Cheng MH, Witzigmann D, Cullis PR. Chander N, et al. Mol Ther Methods Clin Dev. 2023 Jun 12;30:235-245. doi: 10.1016/j.omtm.2023.06.005. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37564393 Free PMC article.

171

Inducible HEK293 AAV packaging cell lines expressing Rep proteins.

Jalšić L, Lytvyn V, Elahi SM, Hrapovic S, Nassoury N, Chahal PS, Gaillet B, Gilbert R. Jalšić L, et al. Mol Ther Methods Clin Dev. 2023 Jul 15;30:259-275. doi: 10.1016/j.omtm.2023.07.002. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37560197 Free PMC article.

172

Prevention of early-onset cardiomyopathy in Dmd exon 52-54 deletion mice by CRISPR-Cas9-mediated exon skipping.

Rok M, Wong TWY, Maino E, Ahmed A, Yang G, Hyatt E, Lindsay K, Fatehi S, Marks R, Delgado-Olguín P, Ivakine EA, Cohn RD. Rok M, et al. Mol Ther Methods Clin Dev. 2023 Jul 17;30:246-258. doi: 10.1016/j.omtm.2023.07.004. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37545481 Free PMC article.

173

Optimizing and developing a scalable, chemically defined, animal component-free lentiviral vector production process in a fixed-bed bioreactor.

Fiol CR, Collignon ML, Welsh J, Rafiq QA. Fiol CR, et al. Mol Ther Methods Clin Dev. 2023 Jul 3;30:221-234. doi: 10.1016/j.omtm.2023.06.011. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37528866 Free PMC article.

174

Goody two plasmids: An optimized transient transfection system for AAV vector production.

Moreno Velasquez SD, Gerstmann E, Grimm D. Moreno Velasquez SD, et al. Mol Ther Methods Clin Dev. 2023 Jul 13;30:191-193. doi: 10.1016/j.omtm.2023.06.010. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37519408 Free PMC article. No abstract available.

175

Novel mutations in U_L24 and gH rescue efficient infection of an HSV vector retargeted to TrkA.

Marzulli M, Hall BL, Zhang M, Goins WF, Cohen JB, Glorioso JC. Marzulli M, et al. Mol Ther Methods Clin Dev. 2023 Jul 3;30:208-220. doi: 10.1016/j.omtm.2023.06.012. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37519407 Free PMC article.

176

Impact of an autophagy-inducing peptide on immunogenicity and protection efficacy of an adenovirus-vectored SARS-CoV-2 vaccine.

Sayedahmed EE, Araújo MV, Silva-Pereira TT, Chothe SK, Elkashif A, Alhashimi M, Wang WC, Santos AP, Nair MS, Gontu A, Nissly R, Francisco de Souza Filho A, Tavares MS, Ayupe MC, Salgado CL, Donizetti de Oliveira Candido É, Leal Oliveira DB, Durigon EL, Heinemann MB, Morais da Fonseca D, Jagannath C, Sá Guimarães AM, Kuchipudi SV, Mittal SK. Sayedahmed EE, et al. Mol Ther Methods Clin Dev. 2023 Jun 27;30:194-207. doi: 10.1016/j.omtm.2023.06.009. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37502665 Free PMC article.

177

CRISPR-Cas9 correction in the DMD mouse model is accompanied by upregulation of Dp71f protein.

Egorova TV, Polikarpova AV, Vassilieva SG, Dzhenkova MA, Savchenko IM, Velyaev OA, Shmidt AA, Soldatov VO, Pokrovskii MV, Deykin AV, Bardina MV. Egorova TV, et al. Mol Ther Methods Clin Dev. 2023 Jun 17;30:161-180. doi: 10.1016/j.omtm.2023.06.006. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37457303 Free PMC article.

178

A method for polyclonal antigen-specific T cell-targeted genome editing (TarGET) for adoptive cell transfer applications.

Palianina D, Di Roberto RB, Castellanos-Rueda R, Schlatter F, Reddy ST, Khanna N. Palianina D, et al. Mol Ther Methods Clin Dev. 2023 Jun 19;30:147-160. doi: 10.1016/j.omtm.2023.06.007. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37448595 Free PMC article.

179

The p53 challenge of hematopoietic stem cell gene editing.

Dorset SR, Bak RO. Dorset SR, et al. Mol Ther Methods Clin Dev. 2023 Jun 12;30:83-89. doi: 10.1016/j.omtm.2023.06.003. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37435043 Free PMC article. Review.

180

Efficacy and muscle safety assessment of fukutin-related protein gene therapy.

Benasutti H, Maricelli JW, Seto J, Hall J, Halbert C, Wicki J, Heusgen L, Purvis N, Regnier M, Lin DC, Rodgers BD, Chamberlain JS. Benasutti H, et al. Mol Ther Methods Clin Dev. 2023 Jun 4;30:65-80. doi: 10.1016/j.omtm.2023.05.022. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37361354 Free PMC article.

181

Bringing gene therapy to regenerative medicine.

Balmayor ER. Balmayor ER. Mol Ther Methods Clin Dev. 2023 Jun 16;30:81-82. doi: 10.1016/j.omtm.2023.05.021. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37361353 Free PMC article. No abstract available.

182

Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6.

Mellen RW, Calabro KR, McCullough KT, Crosson SM, Cova A, Fajardo D, Xu E, Boye SL, Boye SE. Mellen RW, et al. Mol Ther Methods Clin Dev. 2023 Jun 1;30:48-64. doi: 10.1016/j.omtm.2023.05.020. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37361352 Free PMC article.

183

Swapping the serotype: A novel helper-dependent adenoviral vector platform for in vivo HSC gene therapy.

Klatt D, Brendel C, Bauer DE. Klatt D, et al. Mol Ther Methods Clin Dev. 2023 Jun 11;30:14-15. doi: 10.1016/j.omtm.2023.05.018. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37332392 Free PMC article. No abstract available.

184

Starburst amacrine cells amplify optogenetic visual restoration through gap junctions.

Katada Y, Kunimi H, Serizawa N, Lee D, Kobayashi K, Negishi K, Okano H, Tanaka KF, Tsubota K, Kurihara T. Katada Y, et al. Mol Ther Methods Clin Dev. 2023 May 12;30:1-13. doi: 10.1016/j.omtm.2023.05.011. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37324975 Free PMC article.

185

Human amnionic progenitor cell secretome mitigates the consequence of traumatic optic neuropathy in a mouse model.

McCartan R, Gratkowski A, Browning M, Hahn-Townsend C, Ferguson S, Morin A, Bachmeier C, Pearson A, Brown L, Mullan M, Crawford F, Tzekov R, Mouzon B. McCartan R, et al. Mol Ther Methods Clin Dev. 2023 Apr 18;29:303-318. doi: 10.1016/j.omtm.2023.04.002. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37359418 Free PMC article.

186

Detection of mutant antigen-specific T cell receptors against multiple myeloma for T cell engineering.

Okada M, Shimizu K, Nakazato H, Yamasaki S, Fujii SI. Okada M, et al. Mol Ther Methods Clin Dev. 2023 May 16;29:541-555. doi: 10.1016/j.omtm.2023.05.014. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37359417 Free PMC article.

187

An AAV capsid increases transduction of striatum and a ChAT promoter allows selective cholinergic neuron transduction.

Santoscoy MC, Espinoza P, De La Cruz D, Mahamdeh M, Starr JR, Patel N, Maguire CA. Santoscoy MC, et al. Mol Ther Methods Clin Dev. 2023 May 9;29:532-540. doi: 10.1016/j.omtm.2023.05.001. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37359416 Free PMC article.

188

Gene therapy for cross-correction of somatic organs and the CNS in mucopolysaccharidosis II in rodents and non-human primates.

Chen N, Ehmann DE, Crooker R, Derakhchan K, Fang X, Felice B, Galbreath EJ, Glaus C, Gu H, Huang Y, Li C, Li X, Liu N, Palmieri K, Simic D, Sypek J, Thompson S, Winkelmann CT, Choi VW. Chen N, et al. Mol Ther Methods Clin Dev. 2023 Mar 28;29:286-302. doi: 10.1016/j.omtm.2023.03.014. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37359415 Free PMC article.

189

CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids.

Afanasyeva TAV, Athanasiou D, Perdigao PRL, Whiting KR, Duijkers L, Astuti GDN, Bennett J, Garanto A, van der Spuy J, Roepman R, Cheetham ME, Collin RWJ. Afanasyeva TAV, et al. Mol Ther Methods Clin Dev. 2023 May 17;29:522-531. doi: 10.1016/j.omtm.2023.05.012. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37305852 Free PMC article.

190

Strong ubiquitous micro-promoters for recombinant adeno-associated viral vectors.

Chai S, Wakefield L, Norgard M, Li B, Enicks D, Marks DL, Grompe M. Chai S, et al. Mol Ther Methods Clin Dev. 2023 May 17;29:504-512. doi: 10.1016/j.omtm.2023.05.013. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37287749 Free PMC article.

191

Medicaid coverage practices for approved gene and cell therapies: Existing barriers and proposed policy solutions.

Allen J, Berry D, Cook F, Hume A, Rouce R, Srirangam A, Wellman J, McCombs C. Allen J, et al. Mol Ther Methods Clin Dev. 2023 May 16;29:513-521. doi: 10.1016/j.omtm.2023.05.015. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37287748 Free PMC article. Review.

192

Production and characterization of an AAV1-VP3-only capsid: An analytical benchmark standard.

Mietzsch M, Liu W, Ma K, Bennett A, Nelson AR, Gliwa K, Chipman P, Fu X, Bechler S, McKenna R, Viner R. Mietzsch M, et al. Mol Ther Methods Clin Dev. 2023 May 9;29:460-472. doi: 10.1016/j.omtm.2023.05.002. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37273903 Free PMC article.

193

Pre-existing immunity does not impair the engraftment of CRISPR-Cas9-edited cells in rhesus macaques conditioned with busulfan or radiation.

Essawi K, Hakami W, Naeem Khan MB, Martin R, Zeng J, Chu R, Uchida N, Bonifacino AC, Krouse AE, Linde NS, Donahue RE, Blobel GA, Gerdemann U, Kean LS, Maitland SA, Wolfe SA, Metais JY, Gottschalk S, Bauer DE, Tisdale JF, Demirci S. Essawi K, et al. Mol Ther Methods Clin Dev. 2023 Apr 20;29:483-493. doi: 10.1016/j.omtm.2023.04.004. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37273902 Free PMC article.

194

Development and validation of a model gene therapy biodistribution assay for AVGN7 using digital droplet polymerase chain reaction.

Rodgers BD, Herring SK, Carias DR, Chen J, Rocha AG. Rodgers BD, et al. Mol Ther Methods Clin Dev. 2023 May 10;29:494-503. doi: 10.1016/j.omtm.2023.05.007. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37273901 Free PMC article.

195

A novel dual-plasmid platform provides scalable transfection yielding improved productivity and packaging across multiple AAV serotypes and genomes.

van Lieshout LP, Rubin M, Costa-Grant K, Ota S, Golebiowski D, Panico T, Wiberg E, Szymczak K, Gilmore R, Stanvick M, Burnham B, Gagnon J, Iwuchukwu I, Yang G, Ghazi I, Meola A, Dickerson R, Thiers T, Mustich L, Hayes A, Rivas I, Lotterhand J, Avila N, McGivney J, Yin J, Kelly T. van Lieshout LP, et al. Mol Ther Methods Clin Dev. 2023 May 8;29:426-436. doi: 10.1016/j.omtm.2023.05.004. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37273900 Free PMC article.

196

Analysis of EVs from patients with advanced pancreatic cancer identifies antigens and miRNAs with predictive value.

Vannini I, Rossi T, Melloni M, Valgiusti M, Urbini M, Passardi A, Bartolini G, Gallio C, Azzali I, Bandini S, Ancarani V, Montanaro L, Frassineti GL, Fabbri F, Rapposelli IG. Vannini I, et al. Mol Ther Methods Clin Dev. 2023 May 11;29:473-482. doi: 10.1016/j.omtm.2023.05.009. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37273899 Free PMC article.

197

Light at the end of the tunnel of Corti.

Hanlon K, Büning H, Géléoc GSG. Hanlon K, et al. Mol Ther Methods Clin Dev. 2023 May 23;29:437-438. doi: 10.1016/j.omtm.2023.05.006. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37273898 Free PMC article. No abstract available.

198

Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-particle resolution.

Münter R, Christensen E, Andresen TL, Larsen JB. Münter R, et al. Mol Ther Methods Clin Dev. 2023 May 11;29:450-459. doi: 10.1016/j.omtm.2023.05.008. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37251983 Free PMC article.

199

Deconvolution of spatial sequencing provides accurate characterization of hESC-derived DA transplants in vivo.

Rájová J, Davidsson M, Avallone M, Hartnor M, Aldrin-Kirk P, Cardoso T, Nolbrant S, Mollbrink A, Storm P, Heuer A, Parmar M, Björklund T. Rájová J, et al. Mol Ther Methods Clin Dev. 2023 May 4;29:381-394. doi: 10.1016/j.omtm.2023.04.008. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37251982 Free PMC article.

200

Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice.

Kida S, Koshimura Y, Yoden E, Yoshioka A, Morimoto H, Imakiire A, Tanaka N, Tanaka S, Mori A, Ito J, Inoue A, Yamamoto R, Minami K, Hirato T, Takahashi K, Sonoda H. Kida S, et al. Mol Ther Methods Clin Dev. 2023 May 12;29:439-449. doi: 10.1016/j.omtm.2023.05.010. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37251981 Free PMC article.

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