"Mol Ther Adv"[Journal] - Search Results

Year	Number of Results
2025	14
2026	154

1

Simultaneous gene engineering of T cells with multiple neoantigen-specific T cell receptors for adoptive cell transfer.

Magna M, Levy L, Csomos K, Dinerman AJ, Hakim AA, Levin N, Kim SP, Zacharakis N, Parikh NB, Bui BD, Bhasin A, Beyer RK, Goff SL, Lowery FJ, Rosenberg SA, Krishna S. Magna M, et al. Mol Ther Adv. 2026 Jul 9;34(3):201810. doi: 10.1016/j.omta.2026.201810. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42569360 Free PMC article.

2

Double activation and low IL-2 manufacturing protocol increase expansion of healthy and patient-derived CAR-Tregs.

Ugolini A, Miceli A, Bercher-Brayer C, Cesarano S, Camisa B, Carulli P, Monti P, Ramirez GA, Bonomi E, Rudilosso L, Dagna L, Cesana D, Greco R, Ciceri F, Bonini C, Doglio M. Ugolini A, et al. Mol Ther Adv. 2026 Jul 9;34(3):201808. doi: 10.1016/j.omta.2026.201808. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42569341 Free PMC article.

3

Erratum: Efficacy and safety of SENS-501, a dual-AAV otoferlin gene therapy, for DFNB9 congenital deafness.

Boudra R, Olivier G, Van Ba CT, Barrot L, Rambeau P, Vaux C, Pierredon S, Joséphine C, Pages A, Broussy A, Harrus AG, Dadak S, Liaudet P, Petit GH, Poujade O, Nevoux J, Lahlou G, Loundon N, Le Bec C, Bousquet E, Plion B, Filipe S, Lecomte MJ, Safieddine S, Petit C, Giese APJ, Desire L. Boudra R, et al. Mol Ther Adv. 2026 Jul 27;34(3):201807. doi: 10.1016/j.omta.2026.201807. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42565089 Free PMC article.

4

Co-delivery of lentiviral vectors and Cas9-containing virus-like particles enables rapid, scalable manufacture of gene-edited CAR T cells.

Ferrara F, Wielgosz MM, Park JJ, Bauler M, Wincek C, Mier N, Su Y, Tang L, Lowe BR, Beard JA, Langfitt DM, Zhou S, Throm RE. Ferrara F, et al. Mol Ther Adv. 2026 Jul 9;34(3):201809. doi: 10.1016/j.omta.2026.201809. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42565078 Free PMC article.

5

Natural killer cell cryopreservation: Influence of composition, temperature, and granules on post-thaw survival and function.

Joules A, Louwagie T, Mallya AS, Hubel A. Joules A, et al. Mol Ther Adv. 2026 Jul 10;34(3):201813. doi: 10.1016/j.omta.2026.201813. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42564981 Free PMC article.

6

Safety and HBsAg reduction with a lentiviral vaccine J-51 in chronic hepatitis B: An investigator-initiated pilot trial.

Song J, Zhang Q, Zhang Y, Shen J, Wei R, Wang K, Shen C, Wang W, Chu M, Zhou F, Liang X, Zhang L, Huang J, Qiu C, Wu H, Zhang W. Song J, et al. Mol Ther Adv. 2026 Jun 18;34(3):201790. doi: 10.1016/j.omta.2026.201790. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42564720 Free PMC article.

7

The potential of gene therapy to rescue telomeric and extratelomeric defects in X-linked dyskeratosis congenita.

Llorente-Arroyo D, Lozano ML, Navarro S, Sastre L, Perona R, Bueren JA, Guenechea G, Meza NW. Llorente-Arroyo D, et al. Mol Ther Adv. 2026 Jul 10;34(3):201811. doi: 10.1016/j.omta.2026.201811. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42564579 Free PMC article.

8

Durable antiviral protection via direct in vivo programming of hematopoietic stem cells.

Ehrhardt A. Ehrhardt A. Mol Ther Adv. 2026 Jul 24;34(3):201814. doi: 10.1016/j.omta.2026.201814. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42542622 Free PMC article. No abstract available.

9

Whole-brain biodistribution of AAV2, AAV8, and AAV-B10 after unilateral intracerebroventricular injection.

Ye D, Kayithi A, Yuan J, Wang I, Xu T, Boisvert M, Paquet ME, Chen H. Ye D, et al. Mol Ther Adv. 2026 Jun 29;34(3):201798. doi: 10.1016/j.omta.2026.201798. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42518515 Free PMC article.

10

Highly potent MyoAAV4A vector reverses GSD III pathology in aged mice and enables long-term muscle disease correction in young adult mice.

Liao KA, Yi H, Zhang H, Eisner W, Young SP, Sun B. Liao KA, et al. Mol Ther Adv. 2026 Jun 30;34(3):201800. doi: 10.1016/j.omta.2026.201800. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42518514 Free PMC article.

11

Tacrolimus-resistant regulatory T cells: Combining the best of both worlds?

Kuypers D. Kuypers D. Mol Ther Adv. 2026 Jul 16;34(3):201805. doi: 10.1016/j.omta.2026.201805. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42502509 Free PMC article. No abstract available.

12

Pharmacokinetic and pharmacodynamic characterization of CD8-targeted lentiviral vector for in vivo CD19-directed CAR-T therapy.

Duback V, Kharkwal SS, Vagin V, Paterson K, Sabbio A, Kim J, Gaikwad A, Green J, Dolinski B, Hlavaty K, Zipp G, Joyce S, Koppes A, Varghese B, Trudeau K, Shah J, Fry T, Elpek KG. Duback V, et al. Mol Ther Adv. 2026 Jul 3;34(3):201803. doi: 10.1016/j.omta.2026.201803. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42502508 Free PMC article.

13

Optimization of AAV tools to target Müller glial cells for retinal gene therapy.

Urrutia-Cabrera D, Huppert G, Chu SWL, Wang L, Nguy CB, Liu CF, Lisowski L, Luu CD, Wang JH, Hung S, Hewitt AW, Huang CL, Edwards T, Martin KR, Wong RCB. Urrutia-Cabrera D, et al. Mol Ther Adv. 2026 Jul 3;34(3):201804. doi: 10.1016/j.omta.2026.201804. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42494983 Free PMC article.

14

Anion-exchange chromatography separates structurally heterogeneous and low-potency particles in adeno-associated virus manufacture.

Tsunaka Y, Makihira H, Kono H, Yang Z, Lyu X, Soth S, Fukuhara M, Shibuya R, Yamaguchi Y, Uchiyama S. Tsunaka Y, et al. Mol Ther Adv. 2026 Jul 10;34(3):201812. doi: 10.1016/j.omta.2026.201812. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42494982 Free PMC article.

15

The development of single-cell lineage tracing technology and its application in immunotherapy.

Wang J, Li Z, Chen J, Li H, Chen F, Tan L, Chen X, Liu S, Zhang W, Shao H. Wang J, et al. Mol Ther Adv. 2026 Jul 2;34(3):201802. doi: 10.1016/j.omta.2026.201802. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42494981 Free PMC article. Review.

16

Microdystrophins partially rescue deficits of duchenne muscular dystrophy iPSC-cardiomyocytes.

Keegan AR, Gonzalez AFGS, Zhang R, Doan H, Torres-Bigio SI, Lau F, Ly KN, Lai EC, Mamuyac LC, Bhimaraju S, Mishra A, Polen MK, Ortiz DS, Lam JH, Eguchi A. Keegan AR, et al. Mol Ther Adv. 2026 Jul 2;34(3):201801. doi: 10.1016/j.omta.2026.201801. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42494980 Free PMC article.

17

AI-engineered AAV capsid enables intravitreal delivery for the treatment of diverse retinal degenerations.

Cui M, Liu H, Cai L, Zhang Q, Yuan L, Gao C, Li C, Xi J, Li Y, Wu C, Zheng Q, Liu L, Chen P, Zhou S, Mou X, Wekselblatt J, Zhang Y, Han L, Ren S. Cui M, et al. Mol Ther Adv. 2026 Jul 9;34(3):201806. doi: 10.1016/j.omta.2026.201806. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42494979 Free PMC article.

18

mRNA delivery to the retina restores REP1 function in choroideremia.

de Lemos L, Antas P, Carvalho C, Castro M, Bruno D, Fonseca AF, Rakshit S, Seabra MC. de Lemos L, et al. Mol Ther Adv. 2026 May 28;34(3):201768. doi: 10.1016/j.omta.2026.201768. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42488178 Free PMC article.

19

AAV vector production in suspension cells using PEI transfection and sodium butyrate with orthogonal assessment of function and quality.

Ravendran S, Fammé S, Noer MG, Skov TW, Mikkelsen NS, Lee Schneller J, Dorset SR, Wolff JH, Møller AMJ, Haslund D, Revenfeld ALS, Holm M, Mogensen TH, Møller BK, Mikkelsen JG, Bak RO. Ravendran S, et al. Mol Ther Adv. 2026 Jun 17;34(3):201787. doi: 10.1016/j.omta.2026.201787. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42438474 Free PMC article.

20

Initial efforts of translational development of AAV-encoded Na_ViPA1 for peripherally targeted analgesia in neuropathic pain.

Itson-Zoske B, Shin SM, Hong SK, Yongsong C, Fan F, Yu H. Itson-Zoske B, et al. Mol Ther Adv. 2026 Jun 19;34(3):201794. doi: 10.1016/j.omta.2026.201794. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42436860 Free PMC article.

21

Next generation AAV-F capsid gene therapy rescues disease pathology in a model of pyruvate dehydrogenase complex deficiency.

Keegan A, Çetin Ö, Chilcott EM, Diaz JA, Eaton S, Waddington SN, Counsell JR, Rahman S, Karda R. Keegan A, et al. Mol Ther Adv. 2026 Jun 15;34(3):201781. doi: 10.1016/j.omta.2026.201781. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42436859 Free PMC article.

22

Antimetabolites synergize with non-genotoxic antibody drug conjugate conditioning in hematopoietic stem cell lentiviral gene therapy.

Okalova J, Alexander JS, Patel SR, Branella GM, Barichello de Quevedo C, Baldwin WH, Prince C, Chandrakasan S, Brown HC, Doering CB, Spencer HT. Okalova J, et al. Mol Ther Adv. 2026 Jun 11;34(3):201780. doi: 10.1016/j.omta.2026.201780. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42436858 Free PMC article.

23

Combining modeling approaches for rapid preclinical evaluation of retinal gene therapy.

Saha K. Saha K. Mol Ther Adv. 2026 Jul 4;34(3):201785. doi: 10.1016/j.omta.2026.201785. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42436857 Free PMC article. No abstract available.

24

Engineering inter-promoter spacing in baculovirus dual-expression systems enhances transcription and reduces rAAV2 empty capsids.

Lan L, Qu Y, Wang J, Wu W, Chen Y, Meng W, Song W, Hou Y, Shi L, Nan H, Xu X. Lan L, et al. Mol Ther Adv. 2026 Jun 23;34(3):201792. doi: 10.1016/j.omta.2026.201792. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42436856 Free PMC article.

25

Gene therapy induces synaptic ribbon maturation, synaptogenesis and vision recovery in an adult dog model of retinal degeneration.

Beckwith-Cohen B, Sun K, Occelli LM, Winkler PA, Somma AT, Montiani-Ferreira F, Marinho LFLP, Schall PZ, Parys M, Yuzbasiyan-Gurkan V, Petersen-Jones SM. Beckwith-Cohen B, et al. Mol Ther Adv. 2026 Jun 12;34(3):201782. doi: 10.1016/j.omta.2026.201782. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42436855 Free PMC article.

26

AAV-norrin gene therapy rescues retinal defects in mice with Norrie disease and oxygen-induced retinopathy.

Dillinger AE, Jaegle H, Fuchs H, Strobel B, Redemann N, Tamm ER. Dillinger AE, et al. Mol Ther Adv. 2026 Jun 24;34(3):201795. doi: 10.1016/j.omta.2026.201795. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42436854 Free PMC article.

27

Sindbis virus self-amplifying replicon is compatible with modified nucleotides mediating expression and vaccine responses in vivo.

Azizi H, Agbayani G, Renner TM, Simard B, Iqbal U, Dudani R, Jia Y, McCluskie MJ, Akache B. Azizi H, et al. Mol Ther Adv. 2026 Jun 18;34(3):201789. doi: 10.1016/j.omta.2026.201789. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42436853 Free PMC article.

28

Rapamycin nanoparticles mitigate anti-AAV antibody formation in a mouse model of ornithine transcarbamylase deficiency.

Vicidomini A, Boisgerault F, Romano G, Guarnaccia C, Vidal P, Collaud F, Soria LR, de Sabbata G, Tedesco N, Lisjak M, Saxena H, Camparini L, Iaconcig A, Moretti L, Skoko N, Ilyinskii P, Brunetti-Pierri N, Bortolussi G, Ronzitti G, Kishimoto TK, Muro AF. Vicidomini A, et al. Mol Ther Adv. 2026 Jun 19;34(3):201793. doi: 10.1016/j.omta.2026.201793. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42436852 Free PMC article.

29

BAG3 gene therapy emerges as a potential treatment for myofibrillar myopathies.

Sherer LA, Kirk JA. Sherer LA, et al. Mol Ther Adv. 2026 Jul 7;34(3):201796. doi: 10.1016/j.omta.2026.201796. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42436851 Free PMC article. No abstract available.

30

HSCT in SAVI: evidence for a curative hematopoietic approach and perspectives for gene therapy.

Cognard J, Viel S, Belot A. Cognard J, et al. Mol Ther Adv. 2026 Jun 30;34(3):201784. doi: 10.1016/j.omta.2026.201784. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42422768 Free PMC article. No abstract available.

31

NK cell immunotherapy after analytic treatment interruption is associated with HIV viral control.

Thron LK, Pumtang-On P, Chang JW, Cantor ME, Gorrell-Brown I, Becklin KL, Quinn ZE, Karim AF, Rendahl AK, Pampusch MS, Casares SA, Vezys V, Moriarity BS, Skinner PJ. Thron LK, et al. Mol Ther Adv. 2026 Jun 11;34(3):201778. doi: 10.1016/j.omta.2026.201778. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42422767 Free PMC article.

32

Intra-CNS AAV9-GBA1 delivery yields species and route of administration differences in safety and transgene expression.

Amaral AC, Grubor B, Gianni D, Koetzner L, Abraham N, Bourque S, Brown D, Chen Y, Chicoine KE, Clarner P, De Giovanni PJ, Hamann S, Kirkland M, Mendes OR, Michael M, Nadella MVP, Nambiar K, Sebalusky J, Zeng W, Xu S, Trapa P, Plowey ED, Tien E, Fikes J, Walsh DM, Hirst WD, Suh J, Glajch KE. Amaral AC, et al. Mol Ther Adv. 2026 Jun 11;34(3):201779. doi: 10.1016/j.omta.2026.201779. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42422766 Free PMC article.

33

Neuron-specific WWOX gene therapy produces dose-dependent, durable rescue in a model of WWOX-related epileptic encephalopathy.

Obeid M, Akkawi R, Repudi S, Singh PK, Abudiab B, Jebara T, Berent A, Brennan T, Weiss Y, Shekh-Ahmad T, Aqeilan RI. Obeid M, et al. Mol Ther Adv. 2026 Jun 18;34(3):201791. doi: 10.1016/j.omta.2026.201791. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42422765 Free PMC article.

34

Mitochondrial transplantation reverses the senescence phenotype of SH-SY5Y cells.

Xu L, Wu Y, Wu W, Li X, Deng R, Zhu H, Mao A, Sun P, Zhang X, Xu W, Wei CJ. Xu L, et al. Mol Ther Adv. 2026 Jun 17;34(3):201788. doi: 10.1016/j.omta.2026.201788. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42422764 Free PMC article.

35

First pilot study of intrastromal rAAV-PAX6 gene therapy suggests improved corneal thickness and transcription correction in aniridic mouse.

Kaad SG, Lengyell TC, Mirjalili Mohanna SZ, Djaksigulova D, Korecki AJ, Simpson EM. Kaad SG, et al. Mol Ther Adv. 2026 Jun 17;34(3):201786. doi: 10.1016/j.omta.2026.201786. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42422763 Free PMC article.

36

Defining the safety and efficacy of liver-directed AAV gene therapy using a human liver tissue-equivalent platform.

Ramamurthy RM, Zheng WT, Wachtman SE, Diaz JH, George SK, Simon T, Zhou Y, Wan M, Lu B, Walker SJ, Bishop CE, Doering CB, Spencer HT, Atala A, Porada CD, Almeida-Porada G. Ramamurthy RM, et al. Mol Ther Adv. 2026 Jun 9;34(3):201772. doi: 10.1016/j.omta.2026.201772. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42405183 Free PMC article.

37

Erratum: High-titer modular retroviral vectors enabled by an antisense cassette design preventing dsRNA formation during virus production.

de Silly RV, Reichenbach P, Irving M. de Silly RV, et al. Mol Ther Adv. 2026 Jun 30;34(3):201797. doi: 10.1016/j.omta.2026.201797. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42405182 Free PMC article.

38

Systematic evaluation of BBB-penetrant AAV capsids in marmosets identifies VCAP-102 as a highly efficient brain-transducing capsid.

Matsuzaki Y, Konno A, Sakamoto K, Uchida Y, Terasaki T, Hirai H. Matsuzaki Y, et al. Mol Ther Adv. 2026 Jun 16;34(3):201783. doi: 10.1016/j.omta.2026.201783. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42405181 Free PMC article.

39

CRISPR-Cas9 screening reveals TM9SF2 knockout as a solution to HEK293 cell aggregation for improved AAV production.

Park S, Shin S, Han G, Won Y, Lee SY, Razafsky D, George H, Lee GM. Park S, et al. Mol Ther Adv. 2026 Jun 11;34(3):201777. doi: 10.1016/j.omta.2026.201777. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42382945 Free PMC article.

40

Intracellular trafficking and nuclear compartmentalization of AAV gene delivery.

Hutchinson TE, Singh RE, Oliver JY, Davis A, Potter M, Hsi-Bell J, Mietzsch M, Bennett A, McKenna R. Hutchinson TE, et al. Mol Ther Adv. 2026 Jun 8;34(3):201770. doi: 10.1016/j.omta.2026.201770. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42382944 Free PMC article.

41

Erratum: Impact of downstream purification process and AAV serotype on protein-impurity clearance.

Béard N, Rouby G, Luc C, Thomas C, Vanthournout K, Dubreuil G, Loukili Z, Santambien P, Carapito C, Fragnoud R. Béard N, et al. Mol Ther Adv. 2026 Jun 22;34(3):201769. doi: 10.1016/j.omta.2026.201769. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42382943 Free PMC article.

42

Omics-guided engineering of CHO cells reveals host targets to support AAV production.

Tzimou K, López-García G, Sipahi MI, Nielsen LK, Lavado-García J. Tzimou K, et al. Mol Ther Adv. 2026 Jun 8;34(3):201771. doi: 10.1016/j.omta.2026.201771. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42382942 Free PMC article.

43

It's what's on the inside that counts: Capsid content as a critical quality attribute for AAV characterization.

Earley LF, Nelson S, Dolinar Češarek A, Dobnik D. Earley LF, et al. Mol Ther Adv. 2026 Jun 18;34(3):201765. doi: 10.1016/j.omta.2026.201765. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42375475 Free PMC article.

44

Epitope mapping of humoral immunogenicity of orvacabtagene autoleucel shows an IgM response with minimal impact on CAR T cellular kinetics.

Liu X, Hu H, Dai Y, Pazos M, Gokemeijer J, Ogasawara K, Stoevesandt O, Stadler V, Mora J, Jawa V. Liu X, et al. Mol Ther Adv. 2026 May 22;34(3):201763. doi: 10.1016/j.omta.2026.201763. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42375474 Free PMC article.

45

Efficacy and safety of SENS-501, a dual-AAV otoferlin gene therapy, for DFNB9 congenital deafness.

Boudra R, Olivier G, Tran Van Ba C, Barrot L, Rambeau P, Vaux C, Pierredon S, Joséphine C, Pages A, Broussy A, Harrus AG, Dadak S, Liaudet P, Petit GH, Poujade O, Nevoux J, Lahlou G, Loundon N, Le Bec C, Bousquet E, Plion B, Filipe S, Lecomte MJ, Safieddine S, Petit C, Giese APG, Desire L. Boudra R, et al. Mol Ther Adv. 2026 May 19;34(3):201762. doi: 10.1016/j.omta.2026.201762. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42318138 Free PMC article.

46

Deep-sequencing-guided library screening and profiling of AAV capsids in the primate retina.

Fouani Y, Blazevic D, Oti M, Becker R, Strobel B, Schönberger T, Stephan M, Wichert S, Reichel FF, Seitz IP, Korbmacher B, Achberger K, Roosz J, Loskill P, Maier U, Michelfelder S, Schön C. Fouani Y, et al. Mol Ther Adv. 2026 May 22;34(3):201764. doi: 10.1016/j.omta.2026.201764. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42318137 Free PMC article.

47

Preclinical efficacy of a gene therapy for CHKB-mediated muscular dystrophy.

Tavasoli M, Alkandari M, Dorighello G, Devitt J, Hagerty L, Damsker J, Hoffman EP, McMaster CR. Tavasoli M, et al. Mol Ther Adv. 2026 May 25;34(3):201766. doi: 10.1016/j.omta.2026.201766. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42292914 Free PMC article.

48

Preclinical evaluation of AAV2 anti-VEGF gene therapy using scFv for choroidal neovascularization.

Xu X, Luo G. Xu X, et al. Mol Ther Adv. 2026 May 28;34(3):201767. doi: 10.1016/j.omta.2026.201767. eCollection 2026 Sep 10. Mol Ther Adv. 2026. PMID: 42292913 Free PMC article.

49

In vivo adenine base editing rescues brain biochemistry and improves motor deficits in a common phenylketonuria variant.

Gautier MK, King K, Han Y, Said H, Alameh MG, Wang X, Hong X, Musunuru K, Ahrens-Nicklas RC. Gautier MK, et al. Mol Ther Adv. 2026 May 14;34(2):201760. doi: 10.1016/j.omta.2026.201760. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42290942 Free PMC article.

50

Lentiviral rescue of UMPS auxotrophy enables drug-free selection and stable vector expression.

Steding H, Dörpmund N, Herbst J, Sauer M, Maetzig T. Steding H, et al. Mol Ther Adv. 2026 May 15;34(2):201761. doi: 10.1016/j.omta.2026.201761. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42290941 Free PMC article.

51

Seroprevalence of anti-AAV neutralizing antibodiesin healthy and retinitis pigmentosa cohorts: A multi-province study in China.

Du J, Jia H, Xie X, Li X, Zhang T, Wu Y, Liu Z, Zhang H, Li Z, Gu W, Tao X, Chen J, Sun J, Li T, Sun X. Du J, et al. Mol Ther Adv. 2026 Apr 28;34(2):201743. doi: 10.1016/j.omta.2026.201743. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42290940 Free PMC article.

52

Efficacy of anti-VEGF single-chain variable fragment AAV-based gene therapy in a laser-induced choroidal neovascularisation mouse model.

Salman A, Griffiths H, Garba K, Lynn SA, Cree AJ, Szczepan M, Jashal CB, Chozhavel T M R, Rao P, Modi K, MacLaren RE, Yuen HM, Lotery AJ. Salman A, et al. Mol Ther Adv. 2026 May 9;34(2):201756. doi: 10.1016/j.omta.2026.201756. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42254804 Free PMC article.

53

U7snRNA-mediated skipping of intron-derived pseudoexons restores full-length DMD expression in patient-derived cell lines.

Beljan JC, Nicolau S, Lin H, Lay JW, Frair EC, Gushchina LV, Weiss RB, Flanigan KM. Beljan JC, et al. Mol Ther Adv. 2026 May 6;34(2):201750. doi: 10.1016/j.omta.2026.201750. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42232222 Free PMC article.

54

Efficient and safe lung gene delivery using AAV6.2FF in neonatal pigs demonstrates pediatric translational potential.

Zielinska N, Howard EL, Yang C, Stevens BAY, Goens MM, Pei Y, Thompson B, Caswell JL, Thebaud B, Wey D, Valverde A, Arroyo LG, Wootton SK. Zielinska N, et al. Mol Ther Adv. 2026 May 14;34(2):201739. doi: 10.1016/j.omta.2026.201739. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42232221 Free PMC article.

55

Impact of downstream purification process and AAV serotype on protein-impurity clearance.

Béard N, Rouby G, Luc C, Thomas C, Vanthournout K, Dubreuil G, Loukili Z, Santambien P, Carapito C, Fragnoud R. Béard N, et al. Mol Ther Adv. 2026 May 6;34(2):201752. doi: 10.1016/j.omta.2026.201752. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42232220 Free PMC article.

56

Extracellular vesicles as biomarkers of disease progression and therapeutic response in patients with spinal muscular atrophy.

Poulin KL, René CA, Smith IC, Vacratsis PO, Burger D, McMillan HJ, Kothary R, Perkins TJ, Warman-Chardon J, Parks RJ. Poulin KL, et al. Mol Ther Adv. 2026 May 13;34(2):201757. doi: 10.1016/j.omta.2026.201757. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42232219 Free PMC article.

57

It takes two: A two-component system to visualize single-molecule HIV-1 infection in vivo.

Dudek AM. Dudek AM. Mol Ther Adv. 2026 May 22;34(2):201758. doi: 10.1016/j.omta.2026.201758. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42222356 Free PMC article. No abstract available.

58

Revisiting viral reprogramming: Measles virus as a robust platform for high-quality iPSC generation.

Moutaoufik MT, Chahine M. Moutaoufik MT, et al. Mol Ther Adv. 2026 May 22;34(2):201759. doi: 10.1016/j.omta.2026.201759. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42222355 Free PMC article. No abstract available.

59

Prevalence of pre-existing neutralizing antibodies to AAV5 and AAV8 in patients with Wilson's disease.

Zheng ZW, Liu H, Guo LY, Fan LN, Liu C, Li FR, Zhou R, Qu L, Wang RM, Xu WQ, Yang GM, Dong B, Dong Y, Wu ZY. Zheng ZW, et al. Mol Ther Adv. 2026 May 9;34(2):201755. doi: 10.1016/j.omta.2026.201755. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42222354 Free PMC article.

60

Topical delivery of a PPARγ agonist enables adipogenic repair of irradiated skin.

Siegel A, Hanumantharao S, Koirala P, Sehgal V, He C, Wu M, Ali Akbari Ghavimi S, Chen Z, Williams Z, Xu X, Liu C, Kozono D, Agarwal S. Siegel A, et al. Mol Ther Adv. 2026 Apr 6;34(2):201732. doi: 10.1016/j.omta.2026.201732. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42211743 Free PMC article.

61

External controls for rare disease drug development: Lessons for emerging and advanced therapeutic modalities.

Hughes SH, Beretich LA, Fuller M, Goodspeed K, Penn M, Valentino LA, McCombs C. Hughes SH, et al. Mol Ther Adv. 2026 May 19;34(2):201754. doi: 10.1016/j.omta.2026.201754. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42211742 Free PMC article.

62

Efficient NK cell transduction with VSV-G-pseudotyped lentiviral vectors.

Järvelä E, Koski J, Jahan F, Tuhkala A, Saari M, Elmadani M, Salokas K, Veltman L, Andersson L, Kerkelä E, Can H, Nyman M, Ylä-Herttuala S, Varjosalo M, Schenkwein D, Paavilainen H, Vettenranta K, Korhonen M, Göös H. Järvelä E, et al. Mol Ther Adv. 2026 May 5;34(2):201749. doi: 10.1016/j.omta.2026.201749. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42211741 Free PMC article.

63

SauCas9-based cell cycle-dependent genome editing via AAV delivery.

Matsugi E, Kishi K, Kishi A, Nagase K, Nigorikawa K, Nomura W. Matsugi E, et al. Mol Ther Adv. 2026 May 6;34(2):201751. doi: 10.1016/j.omta.2026.201751. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42211740 Free PMC article.

64

Transcript-independent quantitative analysis of RNA cap structures by LC-MS/MS.

Kilburn R, Gomez RC, Evans S. Kilburn R, et al. Mol Ther Adv. 2026 May 1;34(2):201748. doi: 10.1016/j.omta.2026.201748. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42205550 Free PMC article.

65

Fourth-generation gene editors: Integration-based genome engineering.

Kincaid AE, Hewitt AW, Kc R. Kincaid AE, et al. Mol Ther Adv. 2026 May 7;34(2):201753. doi: 10.1016/j.omta.2026.201753. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42200190 Free PMC article. Review.

66

Continuous production of recombinant adeno-associated virus in the insect cell/baculovirus expression vector system.

Joseph J, Destro F, Yuan A, Antov D, Chen W, Song S, Theriault M, Yuan C, Sansom A, Lundin C, Burns T, Hamed HA, Morales P, Fan JX, Rodriguez AR, Sichuma S, McCracken C, Neufeld C, Wolfrum JM, Srinivasan P, Barone PW, Sinskey AJ, Braatz RD, Springs SL. Joseph J, et al. Mol Ther Adv. 2026 Apr 29;34(2):201744. doi: 10.1016/j.omta.2026.201744. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42179677 Free PMC article.

67

Efficient, fratricide free non-viral engineering of CD70-targeted CAR NK cells for hematologic and solid tumor immunotherapy.

Chang JW, Krueger J, Folsom TD, Skeate JG, Stelljes EM, Lahr WS, Slipek NJ, Pomeroy EJ, Webber BR, Moriarity BS. Chang JW, et al. Mol Ther Adv. 2026 Apr 9;34(2):201734. doi: 10.1016/j.omta.2026.201734. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42179676 Free PMC article.

68

A new fully closed automated banking process for healthy autologous cells to standardize the starting material for CAR T manufacturing.

Xu Z, Xu Y, Ding W, Chen J, He Y, Chen J, Sun Y, Guo X, Qian Q. Xu Z, et al. Mol Ther Adv. 2026 Apr 30;34(2):201745. doi: 10.1016/j.omta.2026.201745. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42179675 Free PMC article.

69

Chromophore-loaded CRALBP restores rod visual cycle in chromophore-deficient mice.

van de Werken RP, Sparrow JR, Tsang SH. van de Werken RP, et al. Mol Ther Adv. 2026 May 12;34(2):201747. doi: 10.1016/j.omta.2026.201747. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42179674 Free PMC article. No abstract available.

70

The dose makes the poison: Mechanisms of cytotoxicity of high-dose AAV vectors.

Flotte TR. Flotte TR. Mol Ther Adv. 2026 May 9;34(2):201746. doi: 10.1016/j.omta.2026.201746. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42170349 Free PMC article. No abstract available.

71

Efficacy of intranasal adenovirus vector vaccine is attenuated by type I IFN-induced NK cell activation.

Nakatani H, Tachibana M, Onishi R, Nakagaki T, Ishii KJ, Shimizu K, Sakurai F, Mizuguchi H. Nakatani H, et al. Mol Ther Adv. 2026 Apr 24;34(2):201742. doi: 10.1016/j.omta.2026.201742. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42164241 Free PMC article.

72

Engineering novel AAV capsids by broadly attenuated and subsequent muscle-specific tropism in mice and NHPs.

Pan Y, Zhong Y, Chen H, Zhang Y, Dai Z, Chen J, Tan K, Chen X, Qiu D, Sheng L, Tan X, Fan Y, Bu Y, Zhou Z, Yang Z, Duan R, Guan M, Gao G, Li H. Pan Y, et al. Mol Ther Adv. 2026 Mar 28;34(2):201725. doi: 10.1016/j.omta.2026.201725. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42147446 Free PMC article.

73

Arrayed dual-gRNA CRISPR screening platform for C9orf72 repeat expansion excision in patient iPSCs.

Arogundade OA, Lam KJK, Brown KA, Jain T, Issagholian-Lewin PO, Huang C, Rae-Hudson T, Briseno K, Wyman SK, Krishnappa N, George CA, O'Dare K, Wilson RHC, van Eijk P, Reed SH, Giannikopoulos P, Clelland CD. Arogundade OA, et al. Mol Ther Adv. 2026 Apr 20;34(2):201741. doi: 10.1016/j.omta.2026.201741. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42147445 Free PMC article.

74

Clinical development of tacrolimus-resistant regulatory T cells to enable simultaneous immunosuppression and immune regulation.

Zarrinrad G, Burkhardt LM, Beltran-Mestres C, Romero-Suárez S, Wagner DL, Schlickeiser S, Stein M, Wendering DJ, Carrera Diaz de la Cebosa IJ, Ehlen L, Hamm F, Kurgan GL, Durek P, Heinrich F, Giri A, Pu Y, Ou K, Hoffmann H, Muench S, Stuewe I, Dolling S, McCallion O, Kaeda J, Kath J, Schulenberg S, Wiese N, Nazari A, Peter L, Picht S, Sánchez-Peña A, Usman O, Brommel C, Turk R, Rettig G, Sturgeon M, Osborne TL, Jacobi A, Friedrich R, Kaufmann MM, Klermund J, Fink S, Templin MF, Roemhild A, Kaiser D, Klein O, Cathomen T, Mashreghi MF, Issa F, Polánsky JK, Volk HD, Schmueck-Henneresse M, Reinke P, Amini L. Zarrinrad G, et al. Mol Ther Adv. 2026 Apr 9;34(2):201735. doi: 10.1016/j.omta.2026.201735. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42147444 Free PMC article.

75

A single-cell multi-omics assay for simultaneous measurement of vector copy number and protein expression in CAR T cells.

Yang Y, Parikh S, Li CY, Murphy L, Patel K, Ayaz Q, Mohiuddin M, Elliott JT, He HJ, He Z, Maragh S, Schroeder B, Fry TJ, Winters A, Wang S. Yang Y, et al. Mol Ther Adv. 2026 Apr 15;34(2):201738. doi: 10.1016/j.omta.2026.201738. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42147443 Free PMC article.

76

The good, the bad, and the stable: Reference genes for preclinical biodistribution studies.

Mackeben K, Müller S, Dolim K, Otteneder MB, Ros F, Fakhiri J. Mackeben K, et al. Mol Ther Adv. 2026 Apr 16;34(2):201740. doi: 10.1016/j.omta.2026.201740. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42147442 Free PMC article.

77

Systemic clinical-grade recombinant decorin reduces disease burden and fibrosis in advanced recessive dystrophic epidermolysis bullosa.

Gretzmeier C, Hang B, Sengle G, Phillips C, Buer B, Fletcher S, Belov A, Bleck G, Collins IJ, Brinckmann J, de Souza MP, Landy H, Nyström A. Gretzmeier C, et al. Mol Ther Adv. 2026 Apr 15;34(2):201737. doi: 10.1016/j.omta.2026.201737. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42147441 Free PMC article.

78

Swine reporter model for preclinical evaluation and characterization of gene delivery vectors.

Campbell JM, Korpela DM, Han H, Zhao S, Webster DA, Nguyen YAH, Koes N, Aune RO, Dagan H, Milliken R, Watts JK, Murthy N, Carlson DF. Campbell JM, et al. Mol Ther Adv. 2026 Apr 3;34(2):201729. doi: 10.1016/j.omta.2026.201729. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137606 Free PMC article.

79

Ferrostatin-1 and hinokitiol supplementation enhance human hematopoietic stem cell expansion in a chemically defined medium.

Li L, Mandal PK. Li L, et al. Mol Ther Adv. 2026 Mar 3;34(2):201711. doi: 10.1016/j.omta.2026.201711. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137605 Free PMC article.

80

Low dose systemic AAV-exendin-4 gene therapy for Prader-Willi syndrome and dietary obesity.

Huang W, Xiao M, Zou X, Mohammed T, Feng Z, Cao L. Huang W, et al. Mol Ther Adv. 2026 Mar 12;34(2):201718. doi: 10.1016/j.omta.2026.201718. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137604 Free PMC article.

81

Optimized AAV vector enables potent therapeutic rescue of inherited glycosylphosphatidylinositol deficiency in mice.

Umeshita S, Imanishi K, Likhite S, Ito M, Takino N, Meyer KC, Kinoshita T, Muramatsu S, Murakami Y. Umeshita S, et al. Mol Ther Adv. 2026 Mar 28;34(2):201724. doi: 10.1016/j.omta.2026.201724. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137603 Free PMC article.

82

Rapid multiplex liver gene-editing in mice using adeno-associated virus 8 or lipid nanoparticles.

Wu D, Bolt I, Tolenaars DW, Duijst S, In Het Panhuis W, Hofstraat SRJ, Zwolsman R, van der Meel R, Paulusma CC, van de Graaf SFJ. Wu D, et al. Mol Ther Adv. 2026 Mar 18;34(2):201720. doi: 10.1016/j.omta.2026.201720. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137602 Free PMC article.

83

Single-cell and spatial transcriptomic analyses of gene therapy-associated retinal inflammation in non-human primates.

Sourd C, Quinn J, John MC, Martinez-Fernandez de la Camara C, Wickramasinghe LC, Attar M, Shamsnajafabadi H, Salman A, Cowley SA, Dendrou CA, MacLaren RE, Cehajic-Kapetanovic J, Xue K. Sourd C, et al. Mol Ther Adv. 2026 Mar 30;34(2):201726. doi: 10.1016/j.omta.2026.201726. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137601 Free PMC article.

84

Epigenetic editing balances TCR suppression and persistence in CAR T cells.

Schönberg PY, Muñoz-Ovalle Á, Saleh HA, Crespo E, Kuhnert R, Michen S, Loureiro L, Temme A, Feldmann A, Buchholz F. Schönberg PY, et al. Mol Ther Adv. 2026 Mar 6;34(2):201712. doi: 10.1016/j.omta.2026.201712. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137600 Free PMC article.

85

Efficient and reliable measles reprogramming platform for the generation of human iPSC.

Vashistha S, Hoffer S, Dade J, Majerus S, Royster A, Caya C, Sharp B, Devaux P. Vashistha S, et al. Mol Ther Adv. 2026 Mar 20;34(2):201723. doi: 10.1016/j.omta.2026.201723. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137599 Free PMC article.

86

Gene therapy induces pigmentation in induced pluripotent stem cell-derived retinal pigment epithelium of OCA1A patients and albino rat eye.

George A, Leigh A, Selzer E, Pfister T, DeYoung C, Villasmil R, Abu-Asab M, Hiriyanna S, Hwang P, Sharma R, Maminishkis A, Li T, Brooks BP. George A, et al. Mol Ther Adv. 2026 Feb 20;34(2):201705. doi: 10.1016/j.omta.2026.201705. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137598 Free PMC article.

87

IL-12-engineered human PSMA-CAR T cells for the treatment of advanced prostate cancer.

Lopez LS, Cui Z, Yamaguchi Y, Murad JP, Yang Z, Zou K, Yang J, Chang WC, Forman SJ, Chan VW, Priceman SJ. Lopez LS, et al. Mol Ther Adv. 2026 Apr 2;34(2):201730. doi: 10.1016/j.omta.2026.201730. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137597 Free PMC article.

88

Erratum: Impact of pre-existing immunity on safety and biodistribution of a single AAV9 vector intrathecal injection in cynomolgus monkeys.

Vono M, Rio TD, Magdaleno E, Loll NR, Jarvis P, Walter C, Kikkawa R, Mansfield K, McBlane F, Sirivelu MP, Ozsolak F, Brees D, Hudry E, Rubic-Schneider T, Tukov FF, Huang G. Vono M, et al. Mol Ther Adv. 2026 Apr 15;34(2):201731. doi: 10.1016/j.omta.2026.201731. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137596 Free PMC article.

89

Autofluorescence imaging reveals the impact of cryopreservation on T cell metabolism and activation response.

Pham DL, Kalluri M, Weaver C, Hsu A, Gillette A, Zhao W, Kearl T, Hematti P, Shah NN, Skala MC. Pham DL, et al. Mol Ther Adv. 2026 Feb 19;34(2):201704. doi: 10.1016/j.omta.2026.201704. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137595 Free PMC article.

90

Low-density lipoproteins in human serum competitively inhibit the binding and entry of vesicular stomatitis virus.

Vandergaast R, Johnson S, Ziegler C, Nenavath GN, Schnebeck L, Narjari R, Russell SJ. Vandergaast R, et al. Mol Ther Adv. 2026 Mar 19;34(2):201721. doi: 10.1016/j.omta.2026.201721. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137594 Free PMC article.

91

Using the nose as a factory to secrete proteins into the lungs or circulation.

Sinadinos A, Bell R, Juarez-Molina CI, Meng C, Castells E, Viegas MA, Gill DR, Hyde SC, Griesenbach U, Alton EWFW. Sinadinos A, et al. Mol Ther Adv. 2026 Apr 6;34(2):201733. doi: 10.1016/j.omta.2026.201733. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137593 Free PMC article.

92

A phase 1 clinical trial of a multi-antigen SARS-CoV DNA vaccine as a booster after dose three spike-based mRNA vaccinations.

Aleman S, Ahlén G, Yan J, Ljungman P, Bergman P, Nordentoft CAB, Appelberg S, Alisjahbana A, Cadossi M, Salati S, Tobin K, Tuvesson O, Tegel H, Gidlund EK, Weber F, Larsson O, Höglund U, Buggert M, Hober S, Frelin L, Mirazimi A, Sällberg M. Aleman S, et al. Mol Ther Adv. 2026 Feb 28;34(2):201710. doi: 10.1016/j.omta.2026.201710. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137592 Free PMC article.

93

Development of bicistronic plasmids and fusion proteins for clinical translation of tumor immune reprogramming.

Yang J, Chen SS, Idnani E, Shannon SR, Luly K, Fabilane CS, Wilson DS, Spangler JB, Tzeng SY, Green JJ. Yang J, et al. Mol Ther Adv. 2026 Feb 28;34(2):201708. doi: 10.1016/j.omta.2026.201708. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137591 Free PMC article.

94

Adeno-associated virus-induced neurotoxicity is prevented by CpG depletion.

Dunn CE, Carvajal BV, Ma J, Castañeda-Hernandez G, Nguyen AV, Jung C, Suriano CM, Boulanger LM. Dunn CE, et al. Mol Ther Adv. 2026 Feb 19;34(2):201703. doi: 10.1016/j.omta.2026.201703. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137590 Free PMC article.

95

Development of in vitro potency assays for AAV-based gene silencing therapies targeting FSHD and CMT1A.

McCoy J, Wallace LM, Zhou B, Price B, Salzman R, Harper SQ. McCoy J, et al. Mol Ther Adv. 2026 Apr 1;34(2):201727. doi: 10.1016/j.omta.2026.201727. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137589 Free PMC article.

96

Erratum: Efficient kidney gene transfer and proximal tubule transduction using self-complementary AAV.cc47 vectors.

Peek JL, Rosales A, Qi J, Feleke L, Menshikh A, Welch RC, Woodard LE, Asokan A, Wilson MH. Peek JL, et al. Mol Ther Adv. 2026 Apr 16;34(2):201736. doi: 10.1016/j.omta.2026.201736. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137588 Free PMC article.

97

Engineered T7 RNA polymerase to improve mRNA capping efficiency and reduce dsRNA generation during in vitro transcription.

Sanjeev A, Hannigan C, Garrity R, Kamalakkannan DS, Clendaniel V, Mantri M, Glass Z, Kasiewicz LN, Leipheimer J, Khan Z, Mizoguchi T, Peshdary V, Summers CR, Beuning PJ, Cheng CJ. Sanjeev A, et al. Mol Ther Adv. 2026 Mar 20;34(2):201722. doi: 10.1016/j.omta.2026.201722. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137587 Free PMC article.

98

High-titer modular retroviral vectors enabled by an antisense cassette design preventing dsRNA formation during virus production.

Vuillefroy de Silly R, Reichenbach P, Irving M. Vuillefroy de Silly R, et al. Mol Ther Adv. 2026 Mar 12;34(2):201719. doi: 10.1016/j.omta.2026.201719. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137586 Free PMC article.

99

Optimizing single molecule, real-time sequencing for enhanced characterization of adeno-associated viral vector genomes.

Manz J, Ruppert R, Haindl M, Hubbuch J, Pschirer J. Manz J, et al. Mol Ther Adv. 2026 Apr 1;34(2):201728. doi: 10.1016/j.omta.2026.201728. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137585 Free PMC article.

100

In-utero cell transplantation for hypophosphatasia with gene-edited hESC-derived MSCs in a murine model.

Kitamura N, Hasegawa A, Takahashi-Nakamura A, Kasahara M, Akutsu H, Sago H, Samura O, Okamoto A, Umezawa A. Kitamura N, et al. Mol Ther Adv. 2026 Feb 13;34(2):201696. doi: 10.1016/j.omta.2026.201696. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137584 Free PMC article.

101

Consequences of human genetic variations in KIAA0319L, encoding adeno-associated virus receptor, on AAV-mediated gene transfer.

Vasquez JM, Mackey HL, Rostami MR, Leopold PL, Crystal RG. Vasquez JM, et al. Mol Ther Adv. 2026 Feb 28;34(2):201709. doi: 10.1016/j.omta.2026.201709. eCollection 2026 Jun 11. Mol Ther Adv. 2026. PMID: 42137583 Free PMC article.

102

Enhanced transcriptional insulation of lentiviral vectors using "sequence-upgraded polyA long terminal repeats" or "supA-LTRs".

Wright J, Alberts BM, Martinez Quiles ML, Guarino A, Chipchase D, Clarkson NG, Mitrophanous KA, Farley DC. Wright J, et al. Mol Ther Adv. 2025 Dec 30;34(1):201654. doi: 10.1016/j.omta.2025.201654. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42460420 Free PMC article.

103

F/HN-pseudotyped lentiviral vector efficiently transduces non-human primate airways with no evidence of relevant toxicity.

Griesenbach U, McLachlan G, Sinadinos A, Cheminay C, Ashour J, Cox J, Schwotzer D, Vyhnal K, Gupta A, Chan M, Meng C, Pineault K, Castells E, Dean RJ, Viegas MA, Boyd AC, Davies JC, Gill DR, Hyde SC, Blanset D, Alton EW. Griesenbach U, et al. Mol Ther Adv. 2025 Dec 26;34(1):201655. doi: 10.1016/j.omta.2025.201655. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42460419 Free PMC article.

104

Pre-existing retinal inflammation exacerbates gene therapy-associated uveitis in more rapidly progressive retinal degeneration.

John MC, Martinez-Fernandez de la Camara C, Salman A, Staurenghi F, Shamsnajafabadi H, McClements ME, Cehajic-Kapetanovic J, Bray A, Fischer MD, Dick AD, MacLaren RE, Xue K. John MC, et al. Mol Ther Adv. 2025 Dec 26;34(1):201652. doi: 10.1016/j.omta.2025.201652. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42460418 Free PMC article.

105

CRISPR-mediated targeting of the LMNA c.745C>T mutation enhances survival and cardiac function in congenital muscular dystrophy.

Gómez-Domínguez D, Epifano C, Hernández I, Vilaplana-Martí B, Martín A, Amarilla-Quintana S, Cesar S, de Molina-Iracheta A, Sena-Esteves M, Sarquella-Brugada G, Pérez de Castro I. Gómez-Domínguez D, et al. Mol Ther Adv. 2025 Dec 26;34(1):201653. doi: 10.1016/j.omta.2025.201653. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42460417 Free PMC article.

106

Designs of the clinical trials aiming at evaluating cell and gene therapy products: A critical appraisal from a literature review.

Biard L, Lévy V, Chevret S; JOIN4ATMP consortium. Biard L, et al. Mol Ther Adv. 2025 Dec 26;34(1):201651. doi: 10.1016/j.omta.2025.201651. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42460416 Free PMC article. Review.

107

Case study of resecabtagene autoleucel in a subject with diffuse cutaneous systemic sclerosis treated in the RESET-SSc trial.

Nunez D, Volkov JR, Little C, Ghosh M, Tsou PS, Furmanak T, Dey P, Tsoi LC, Zahn C, Bogle R, Cai Y, Fox J, Stadanlick J, Werner M, Vorndran Z, Ishikawa L, Ellis A, Williams J, Cicarelli J, Flannagan S, Kobulsky D, Lam Q, Schmitt C, Nezhad F, Thompson D, Braccia D, Rivera TG, Tummala R, Gudjonsson JE, Ross C, Binder G, Chang D, Basu S, Khanna D. Nunez D, et al. Mol Ther Adv. 2025 Dec 26;34(1):201663. doi: 10.1016/j.omta.2025.201663. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42157967 Free PMC article.

108

Sinbaglustat is efficacious in GM2 gangliosidosis primarily through inhibition of GBA2 rather than GCS.

Steiner MA, Vaillant C, Garzotti M, Gulinello M, VanBrakle ML, Ryge J, Mühlemann A, Dobrenis K, Welford RWD. Steiner MA, et al. Mol Ther Adv. 2025 Dec 26;34(1):201658. doi: 10.1016/j.omta.2025.201658. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42157966 Free PMC article.

109

Gene therapy rescues cone function in opn1mw ^-/- /opn1sw ^-/- /Nrl ^-/- mice, an all-cone model of blue cone monochromacy.

Cahill ME, Chmelik K, Ashcraft ME, Brothers BA, Guan T, Nguyen J, Puja A, Eminhizer M, Shaw LM, Barbera RJ, Sechrest ER, Du J, Deng WT. Cahill ME, et al. Mol Ther Adv. 2025 Dec 26;34(1):201657. doi: 10.1016/j.omta.2025.201657. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42157965 Free PMC article.

110

Role of viral protein ratio in the structure and separation of empty and full adeno-associated virus capsids: A molecular dynamics study.

Valiya Parambathu A, Kumar V, Khanal O, Lenhoff AM. Valiya Parambathu A, et al. Mol Ther Adv. 2025 Dec 26;34(1):201656. doi: 10.1016/j.omta.2025.201656. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42157964 Free PMC article.

111

Global age-related seroprevalence for adeno-associated virus serotype 9 immunoglobulin G.

Lo Bianco C, Chen CW, Jeeninga RE, Vietsch EE, van Olden RW. Lo Bianco C, et al. Mol Ther Adv. 2025 Dec 26;34(1):201662. doi: 10.1016/j.omta.2025.201662. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42157963 Free PMC article.

112

Development of a secretable frataxin for enhanced efficacy in treating Friedreich's Ataxia.

DuBreuil DM, Fleming M, Parikh Y, Woo M, Bu J, Ayloo S, Langohr IM, Bangari DS, Mueller C, Ramachandran S. DuBreuil DM, et al. Mol Ther Adv. 2025 Dec 26;34(1):201661. doi: 10.1016/j.omta.2025.201661. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42157962 Free PMC article.

113

Normothermic perfusion of human livers for profiling lentiviral vector pharmacokinetics and transduction.

Nicholls BRM, Johnson D, Kulkarni A, Raposo RAS, Mitrophanous KA, Coussios CC, Carlisle RC. Nicholls BRM, et al. Mol Ther Adv. 2025 Dec 26;34(1):201660. doi: 10.1016/j.omta.2025.201660. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42157961 Free PMC article.

114

MicroRNA-based targeting strategies enable AAV-mediated, tissue-selective gene expression in adipose tissue and skeletal muscle of mice.

Blahetek G, Abele S, Schmierer E, Matera D, Geisbauer A, Lempp C, Schönberger T, Holdenried-Krafft S, Gross P, Strobel B. Blahetek G, et al. Mol Ther Adv. 2025 Dec 26;34(1):201664. doi: 10.1016/j.omta.2025.201664. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42157960 Free PMC article.

115

RAAVioli: A comprehensive approach to characterizing AAV vector integrations and rearrangements.

Cipriani C, Rudilosso L, Dalwadi DA, Masseroli M, Grompe M, Montini E, Calabria A, Cesana D. Cipriani C, et al. Mol Ther Adv. 2025 Dec 26;34(1):201659. doi: 10.1016/j.omta.2025.201659. eCollection 2026 Mar 12. Mol Ther Adv. 2025. PMID: 42157959 Free PMC article.

116

Advancing AADC deficiency therapy through MR-guided multisite delivery of AAV2-hAADC to dopaminergic and serotonergic pathways in the brainstem.

Samaranch L, Neudorfer C, Rocco MT, O'Brien A, Munjal V, McFarland N, Travis M, Taunque AS, Ceyhan KE, Dandamudi A, Karkhah A, Nimmo A, Bringas J, Akhter AS, Youngblood B, Mattox L, Richardson RM, Forsayeth JR, Lonser RR, Bankiewicz KS. Samaranch L, et al. Mol Ther Adv. 2026 Jan 12;34(1):201670. doi: 10.1016/j.omta.2026.201670. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137299 Free PMC article.

117

Productivity and genetic stability of a novel baculovirus vector for multigene expression from independent transgene loci.

de Jong LA, Schrama M, Willems J, de Wilde AH, Geertsema C, Henkens M, Martens DE, Sanders BP, van Oers MM, Pijlman GP. de Jong LA, et al. Mol Ther Adv. 2026 Jan 9;34(1):201668. doi: 10.1016/j.omta.2026.201668. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137298 Free PMC article.

118

Erratum: CRISPR/methotrexate-integrated strategy for TCR-T cell engineering with reduced chromosome 14 loss.

Xu J, Shen L, Chen Z, Zhang C, Ding X, He Q, Zhou X, Jiang P, Liu J, Li L, Fang J, Li F, Wan L, Ding X, Yang Y, Wang X, Wang P, Song X, Zhang Y. Xu J, et al. Mol Ther Adv. 2026 Jan 23;34(1):201676. doi: 10.1016/j.omta.2026.201676. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137297 Free PMC article.

119

In vivo HSC gene therapy enables sustained eCD4-Ig expression for SIV prevention.

Li C, Anderson AK, Kuhlmann AS, Nelson V, Germond A, Wang H, Georgakopoulou A, Gil S, Martinez-Reyes J, Riker A, Raman SS, Kim J, Ng P, Palmer D, Alpert MD, Skamangas N, Bailey C, Ou T, Fennessey CM, Farzan M, Jerome KR, Keele BF, Kiem HP, Lieber A, Bui JK. Li C, et al. Mol Ther Adv. 2026 Feb 26;34(1):201683. doi: 10.1016/j.omta.2026.201683. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137296 Free PMC article.

120

Voluntary running sustains the correction of inflammation-related gene expression conferred by AAV gene therapy in mdx mice.

Yuan C, Hamm SE, Mack DL, Dupont JB, Grange RW. Yuan C, et al. Mol Ther Adv. 2026 Jan 29;34(1):201679. doi: 10.1016/j.omta.2026.201679. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137295 Free PMC article.

121

Comparative pharmacokinetic derivations for axicabtagene ciloleucel and brexucabtagene autoleucel in the treatment of B cell malignancies.

Salunkhe SS, Nater J, Schweitzer C, Mao D, Dong J, Venkkataram R, Xue A, Filosto S, Mattie M, Shen RR. Salunkhe SS, et al. Mol Ther Adv. 2026 Feb 7;34(1):201686. doi: 10.1016/j.omta.2026.201686. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137294 Free PMC article.

122

CSF and plasma neuro-biomarker dynamics after gene therapy for cerebral adrenoleukodystrophy.

Lund TC, Gupta AO, Kemp S, Loes DJ, Nascene DR, Orchard PJ. Lund TC, et al. Mol Ther Adv. 2026 Feb 18;34(1):201702. doi: 10.1016/j.omta.2026.201702. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137293 Free PMC article.

123

Autophagy activation via BAG3 gene therapy improves phenotype in a mouse model of LGMD1A.

Ozes B, Tong L, Moss K, Myers M, Morrison L, Son E, Vetter TA, Sahenk Z. Ozes B, et al. Mol Ther Adv. 2026 Feb 7;34(1):201687. doi: 10.1016/j.omta.2026.201687. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137292 Free PMC article.

124

Preclinical evaluation of INS1201 AAV9-micro-dystrophin via CSF administration as a potential therapy for Duchenne muscular dystrophy.

Thomsen G, Kaspar A, Ferraiuolo L, Chu B, Garcia VJ, Weiss R, Slaiwa T, Casanova-Vallve N, Cano R, Hurley E, Julazadeh H, Bautista H, Renteria A, Lee M, Rollins J, McAlonis-Downes M, Arnold WD, TreDenick T, Hsu R, Bayless LE, Kozial C, Forespring C, Molony M, Fraser M, Zhou J, Yao J, Bremner SN, Ward SR, Schenk S, Yoshikawa T, Moriyama A, Tsusaki H, Varma S, Kruger M, Stahl M, Kaspar B. Thomsen G, et al. Mol Ther Adv. 2026 Feb 20;34(1):201707. doi: 10.1016/j.omta.2026.201707. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137291 Free PMC article.

125

Systems vaccinology analysis of saRNA immunization identifies an acute innate immune signature correlated with adaptive immunity.

Elliott T, Wang Z, Bonduelle O, Evans A, Day S, McFarlane LR, de Bernard S, Alves K, Nourikyan J, Wokam M, Pollock K, Cheeseman HM, Combadiere B, Shattock RJ, Tregoning JS. Elliott T, et al. Mol Ther Adv. 2026 Feb 20;34(1):201706. doi: 10.1016/j.omta.2026.201706. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137290 Free PMC article.

126

CSF GAG non-reducing ends in MPS IH after peripheral and brain-penetrating therapies: A model comparing IV non-targeted ERT and HCT.

Lund TC, Wagner JE, Long S, Gupta AO, Orchard PJ, Braunlin E, Whitley CB, Eisengart JB. Lund TC, et al. Mol Ther Adv. 2026 Feb 13;34(1):201697. doi: 10.1016/j.omta.2026.201697. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137289 Free PMC article.

127

A comparison of two manufacturing methods in the phase I COBALT study of CD19CAR T for LBCL.

Roddie C, Dias J, Weng-Kit Cheung G, O'Reilly MA, Abbasian M, Cadinanos-Garai A, Vispute K, Bosshard-Carter L, Mitsikakou M, Mehra V, Roddy H, Hartley JA, Mahmoud NG, Ensell L, Patel Y, Marzolini MAV, Farzaneh F, Himoudi N, Syed F, Popova B, Lopes A, Day A, Lowdell MW, Peggs KS. Roddie C, et al. Mol Ther Adv. 2026 Feb 13;34(1):201695. doi: 10.1016/j.omta.2026.201695. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137288 Free PMC article.

128

Interrogating the dependency of AAV capsids on AAVR for retinal transduction.

Hinsch VG, Kellish PC, Xu EM, Fajardo D, McLaughlin KR, Kiel KN, Peterson JJ, Friend HL, Boye SL, Boye SE. Hinsch VG, et al. Mol Ther Adv. 2026 Jan 22;34(1):201677. doi: 10.1016/j.omta.2026.201677. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137287 Free PMC article.

129

Advancing AAV technology-From promise to product.

Hordeaux J, Keeler AM, Stone D. Hordeaux J, et al. Mol Ther Adv. 2026 Jan 20;34(1):201672. doi: 10.1016/j.omta.2026.201672. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137286 Free PMC article. No abstract available.

130

Impact of physical and chemical parameters on spinoculation for chimeric antigen receptor T cell manufacturing using a quality-by-design approach.

Silva Couto P, Stibbs DJ, Sanchez BC, Springuel P, McLean S, Schultz U, Effenberger M, Takeuchi Y, Rafiq QA. Silva Couto P, et al. Mol Ther Adv. 2026 Feb 10;34(1):201691. doi: 10.1016/j.omta.2026.201691. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137285 Free PMC article.

131

Vortex-actuated pre-enrichment accelerates human regulatory T cell sorting and improves early fitness.

Ho QY, Hashimoto H, Hester J, Issa F. Ho QY, et al. Mol Ther Adv. 2026 Jan 9;34(1):201667. doi: 10.1016/j.omta.2026.201667. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137284 Free PMC article.

132

Elective preterm birth for earlier spinal muscular atrophy treatment.

Dangouloff T, Chantraine F, Hennuy N, Rigo V, Tribolet S, Brande LV, Servais L. Dangouloff T, et al. Mol Ther Adv. 2026 Jan 19;34(1):201673. doi: 10.1016/j.omta.2026.201673. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137283 Free PMC article.

133

Humanized mice enable in vivo evaluation of engineered plasma cell biology and therapeutic function.

Hill TF, Helmers AE, Yu-Hong Cheng R, Singh S, Homad LJ, Narvekar P, Asher GD, Camp ND, Suchland ER, Ott AR, Hale M, Thouvenel CD, Stoffers CM, Lachkar S, McGuire AT, Rawlings DJ, James RG. Hill TF, et al. Mol Ther Adv. 2026 Jan 9;34(1):201666. doi: 10.1016/j.omta.2026.201666. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137282 Free PMC article.

134

First successful allogeneic hematopoietic stem cell transplantation in STING-associated vasculopathy of infancy-A case report.

Yu U, Song A, Luo C, Luo C, Qin X, Huang X, Wang X, Lin Y, Zhou C, Zhang M, Chen J, Wang X. Yu U, et al. Mol Ther Adv. 2026 Feb 16;34(1):201699. doi: 10.1016/j.omta.2026.201699. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137281 Free PMC article.

135

Immune shielding of viral vectors via capsid engineering with genetically encoded zwitterionic peptides.

Lu SC, Ma OX, Anwar AA, Ratkov D, Bhandari J, Montiel-Garcia DJ, Hansen MJ, Barry ME, Reddy VS, Barry MA. Lu SC, et al. Mol Ther Adv. 2026 Feb 13;34(1):201694. doi: 10.1016/j.omta.2026.201694. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137280 Free PMC article.

136

Overcoming lentiviral delivery limitations in hard-to-transduce suspension cells for genome-wide CRISPR screening.

Napoleone A, Sebastian IR, De Marco F, Molin A, Hussein M, Kramer L, Jostock T, Kelly T, Borth N. Napoleone A, et al. Mol Ther Adv. 2026 Jan 27;34(1):201678. doi: 10.1016/j.omta.2026.201678. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137279 Free PMC article.

137

Rational engineering of the P5 TRS-mimic site and REP78/68 start codon yields promoter variants that improve rAAV purity while maintaining high titers.

Winston SM, Brimble MA, Wiggins KB, Reeves IL, Spence Y, Fan B, Tan S, Morton CL, Cheng PH, Spence MY, Throm RE, Thomas PG, Gray JT, Crawford JC, Piras BA, Davidoff AM. Winston SM, et al. Mol Ther Adv. 2026 Jan 29;34(1):201680. doi: 10.1016/j.omta.2026.201680. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137278 Free PMC article.

138

Disease models and AAV therapies of PKP2-induced arrhythmogenic cardiomyopathy.

Geerlings L, Carreras YR, Vermeer MCSC. Geerlings L, et al. Mol Ther Adv. 2026 Feb 9;34(1):201688. doi: 10.1016/j.omta.2026.201688. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137277 Free PMC article. Review.

139

CD19 CAR T cell therapy in systemic sclerosis: A case study that reaffirms the promise of cell therapy to treat autoimmune disease.

Riley JL. Riley JL. Mol Ther Adv. 2026 Feb 17;34(1):201684. doi: 10.1016/j.omta.2026.201684. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137276 Free PMC article. No abstract available.

140

Lipid nanoparticles for cell and gene therapy.

Han X, Shi Q, Mitchell MJ. Han X, et al. Mol Ther Adv. 2026 Jan 20;34(1):201671. doi: 10.1016/j.omta.2026.201671. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137275 Free PMC article. No abstract available.

141

Tracking HIV-1 DNA fate from cell culture to humanized mice tissues.

Diana JS, Tatirovsky E, Tomasini C, Cuche C, Ay S, Scoca V, Cavazzana M, Di Santo JP, Di Nunzio F. Diana JS, et al. Mol Ther Adv. 2026 Feb 10;34(1):201690. doi: 10.1016/j.omta.2026.201690. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137274 Free PMC article.

142

Manufacture of adeno-associated virus vectors by a novel human-derived cell line HAT and comprehensive evaluation of the vectors.

Tsunaka Y, Fukuhara M, Shimojo S, Matsushita A, Maruno T, Soth S, Nishiumi H, Vergara Rocafort MA, Kuwahara T, Matsumoto K, Shibata K, Nakatsuka R, Asahina R, Mizukado S, Fukai Y, Togashi T, Baatartsogt N, Takeda K, Kuno A, Kashiwakura Y, Yamaguchi Y, Nakamura K, Hirai Y, Hirai H, Ohmori T, Omasa T, Uchiyama S. Tsunaka Y, et al. Mol Ther Adv. 2026 Feb 14;34(1):201700. doi: 10.1016/j.omta.2026.201700. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137273 Free PMC article.

143

Capsid-engineered AAV vector overcomes a key intracellular barrier and efficiently transduces spiral ganglion neurons in adult mice.

Marx J, Huang P, Sutter SO, Ertelt M, Kaiser O, Harre J, Schott J, Wilkes J, Neek-John P, Rossi A, Warnecke A, Schoeder CT, Schambach A, Staecker H, Büning H. Marx J, et al. Mol Ther Adv. 2026 Jan 13;34(1):201669. doi: 10.1016/j.omta.2026.201669. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137272 Free PMC article.

144

Deciphering the role and contribution of AAV immune responses to DRG toxicity.

Hordeaux J, Chan YK. Hordeaux J, et al. Mol Ther Adv. 2026 Feb 4;34(1):201675. doi: 10.1016/j.omta.2026.201675. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137271 Free PMC article. No abstract available.

145

Chemistry-mediated synthetic virus-cell interaction as a generic approach to enhance gene delivery to mammalian cells.

Tannir R, Chan L, Grzywa TM, Arevalo O, Neeser A, Kahn S, Cozzone A, Ramamurthi A, Olenick L, Mason NJ, Liu X, Ma L. Tannir R, et al. Mol Ther Adv. 2026 Jan 3;34(1):201665. doi: 10.1016/j.omta.2025.201665. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137270 Free PMC article.

146

Design and initial characterization of a novel mini-promoter for gene therapies targeting the central nervous system.

Chauhan M, Daugherty AL, Khadir FE, Duzenli OF, Hoffman A, Wan O, Tinklenberg JA, Kang PB, Aslanidi G, Pacak CA. Chauhan M, et al. Mol Ther Adv. 2026 Jan 29;34(1):201681. doi: 10.1016/j.omta.2026.201681. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137269 Free PMC article.

147

RT-qPCR and split-luciferase assays enable batch standardization and analysis of engineered virus-like particle transduction.

Nicosia L, Murray JB, Collins E, Lonetti L, Harrison PT, Scallan MF. Nicosia L, et al. Mol Ther Adv. 2026 Feb 9;34(1):201685. doi: 10.1016/j.omta.2026.201685. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137268 Free PMC article.

148

The first oral relaxin receptor RXFP1 agonist for heart failure treatment: Translational studies from non-human primates to humans.

Ryberg E, Bergström F, Sten S, Olsson J, Lal M, Connolly K, Matsson E, Åstrand M, Althage M, Hartman RJG, Mohamed E, Fjellstrom O, Pettersen D, George RT, Whittaker A, Kujacic M, Gabrielsen A, Rosenmeier JB. Ryberg E, et al. Mol Ther Adv. 2026 Jan 19;34(1):201674. doi: 10.1016/j.omta.2026.201674. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137267 Free PMC article.

149

Chromophore-loaded CRALBP mutant proteins restore rod function in chromophore-deficient mice.

Kolesnikov AV, Aeschimann W, Cullity J, Halabi M, Kiser PD, Stocker A, Kefalov VJ. Kolesnikov AV, et al. Mol Ther Adv. 2026 Feb 17;34(1):201701. doi: 10.1016/j.omta.2026.201701. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137266 Free PMC article.

150

Spatial mapping of barcoded, brain-tropic AAVs using multiplexed RNA in situ hybridization.

Fouani Y, Oti M, Jarosch S, Blazevic D, Duechs MJ, Ketterer S, Becker R, Abele S, Strobel B, Schoenberger T, Alanis-Lobato G, Rust W, Schoen C, Klugmann M, Maier U, Doerr J, Michelfelder S. Fouani Y, et al. Mol Ther Adv. 2026 Feb 12;34(1):201693. doi: 10.1016/j.omta.2026.201693. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137265 Free PMC article.

151

Plasmid DNA vaccines encapsulated in lipid nanoparticles elicit STING-dependent type 1 interferon release.

Moseman JE, Brinkman HM, Choe JA, Shim D, Burger JC, Ferraresso F, Rastogi I, Kastrup CJ, Murphy WL, McNeel DG. Moseman JE, et al. Mol Ther Adv. 2026 Feb 16;34(1):201698. doi: 10.1016/j.omta.2026.201698. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137264 Free PMC article.

152

DNA damage/p53, innate immune, and unfolded protein responses are activated in primate liver after toxic, high-dose AAV-SMN1 delivery.

Moeini P, Bilbao-Arribas M, Guruceaga E, Torrens-Baile J, Lanz TA, Whiteley LO, Aragón T, Unzu C, González-Aseguinolaza G. Moeini P, et al. Mol Ther Adv. 2026 Jan 30;34(1):201682. doi: 10.1016/j.omta.2026.201682. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137263 Free PMC article.

153

Universal strategy to generate lentiviral vectors encoding lethal proteins.

Versier B, Soundiramourty A, Manchon E, Ravassard P, Charron D, Christen Y, Mallet J, Al-Daccak R, Serguera C. Versier B, et al. Mol Ther Adv. 2026 Feb 7;34(1):201689. doi: 10.1016/j.omta.2026.201689. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137262 Free PMC article.

154

Modulating viscosity improves lentiviral transduction of NK cells: A simple solution to a persistent problem.

Bjelica M, Benyoucef A, Romero H, Beland K, Gagnon E, Haddad E. Bjelica M, et al. Mol Ther Adv. 2026 Feb 10;34(1):201692. doi: 10.1016/j.omta.2026.201692. eCollection 2026 Mar 12. Mol Ther Adv. 2026. PMID: 42137261 Free PMC article.

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