Barzel A - Search Results

1

Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model.

Porro F, Bortolussi G, Barzel A, De Caneva A, Iaconcig A, Vodret S, Zentilin L, Kay MA, Muro AF. Porro F, et al. Among authors: barzel a. EMBO Mol Med. 2017 Oct;9(10):1346-1355. doi: 10.15252/emmm.201707601. EMBO Mol Med. 2017. PMID: 28751579 Free PMC article.

2

Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases.

De Caneva A, Porro F, Bortolussi G, Sola R, Lisjak M, Barzel A, Giacca M, Kay MA, Vlahoviček K, Zentilin L, Muro AF. De Caneva A, et al. Among authors: barzel a. JCI Insight. 2019 Jun 18;5(15):e128863. doi: 10.1172/jci.insight.128863. JCI Insight. 2019. PMID: 31211694 Free PMC article.

3

Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice.

Lisjak M, De Caneva A, Marais T, Barbon E, Biferi MG, Porro F, Barzel A, Zentilin L, Kay MA, Mingozzi F, Muro AF. Lisjak M, et al. Among authors: barzel a. Front Genome Ed. 2022 Mar 11;4:785698. doi: 10.3389/fgeed.2022.785698. eCollection 2022. Front Genome Ed. 2022. PMID: 35359664 Free PMC article.

4

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

Barzel A, Paulk NK, Shi Y, Huang Y, Chu K, Zhang F, Valdmanis PN, Spector LP, Porteus MH, Gaensler KM, Kay MA. Barzel A, et al. Nature. 2015 Jan 15;517(7534):360-4. doi: 10.1038/nature13864. Epub 2014 Oct 29. Nature. 2015. PMID: 25363772 Free PMC article.

5

A universal system to select gene-modified hepatocytes in vivo.

Nygaard S, Barzel A, Haft A, Major A, Finegold M, Kay MA, Grompe M. Nygaard S, et al. Among authors: barzel a. Sci Transl Med. 2016 Jun 8;8(342):342ra79. doi: 10.1126/scitranslmed.aad8166. Sci Transl Med. 2016. PMID: 27280686 Free PMC article.

6

Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia.

Chandler RJ, Venturoni LE, Liao J, Hubbard BT, Schneller JL, Hoffmann V, Gordo S, Zang S, Ko CW, Chau N, Chiang K, Kay MA, Barzel A, Venditti CP. Chandler RJ, et al. Among authors: barzel a. Hepatology. 2021 Jun;73(6):2223-2237. doi: 10.1002/hep.31570. Epub 2021 May 21. Hepatology. 2021. PMID: 32976669 Free PMC article.

7

Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency.

Borel F, Tang Q, Gernoux G, Greer C, Wang Z, Barzel A, Kay MA, Shultz LD, Greiner DL, Flotte TR, Brehm MA, Mueller C. Borel F, et al. Among authors: barzel a. Mol Ther. 2017 Nov 1;25(11):2477-2489. doi: 10.1016/j.ymthe.2017.09.020. Epub 2017 Sep 25. Mol Ther. 2017. PMID: 29032169 Free PMC article.

8

New Horizons for Immune Gene Therapy.

Barzel A, Flotte TR. Barzel A, et al. Hum Gene Ther. 2019 Apr;30(4):379-380. doi: 10.1089/hum.2019.29085.aba. Hum Gene Ther. 2019. PMID: 30950655 No abstract available.

9

MCP-1/CCR2 axis inhibition sensitizes the brain microenvironment against melanoma brain metastasis progression.

Pozzi S, Scomparin A, Ben-Shushan D, Yeini E, Ofek P, Nahmad AD, Soffer S, Ionescu A, Ruggiero A, Barzel A, Brem H, Hyde TM, Barshack I, Sinha S, Ruppin E, Weiss T, Madi A, Perlson E, Slutsky I, Florindo HF, Satchi-Fainaro R. Pozzi S, et al. Among authors: barzel a. JCI Insight. 2022 Sep 8;7(17):e154804. doi: 10.1172/jci.insight.154804. JCI Insight. 2022. PMID: 35980743 Free PMC article.

10

Immune Gene Therapy and the International Conference on Lymphocyte Engineering (ICLE 2018).

Barzel A. Barzel A. Hum Gene Ther. 2018 Jun;29(6):vii-ix. doi: 10.1089/hum.2018.29069.aba. Hum Gene Ther. 2018. PMID: 29902085 No abstract available.

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