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351 results

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Page 1
Base-editing corrects metabolic abnormalities in a humanized mouse model for glycogen storage disease type-Ia.
Arnaoutova I, Aratyn-Schaus Y, Zhang L, Packer MS, Chen HD, Lee C, Gautam S, Gregoire FM, Leboeuf D, Boule S, Fernandez TP, Huang V, Cheng LI, Lung G, Bannister B, Decker J, Leete T, Shuang LS, Bock C, Kothiyal P, Grayson P, Mok KW, Quinn JJ, Young L, Barrera L, Ciaramella G, Mansfield BC, Chou JY. Arnaoutova I, et al. Among authors: barrera l. Nat Commun. 2024 Nov 10;15(1):9729. doi: 10.1038/s41467-024-54108-1. Nat Commun. 2024. PMID: 39523369 Free PMC article.
Rationally Designed Base Editors for Precise Editing of the Sickle Cell Disease Mutation.
Chu SH, Packer M, Rees H, Lam D, Yu Y, Marshall J, Cheng LI, Lam D, Olins J, Ran FA, Liquori A, Gantzer B, Decker J, Born D, Barrera L, Hartigan A, Gaudelli N, Ciaramella G, Slaymaker IM. Chu SH, et al. Among authors: barrera l. CRISPR J. 2021 Apr;4(2):169-177. doi: 10.1089/crispr.2020.0144. CRISPR J. 2021. PMID: 33876959
Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing.
Zhang X, Zhao W, Nguyen GN, Zhang C, Zeng C, Yan J, Du S, Hou X, Li W, Jiang J, Deng B, McComb DW, Dorkin R, Shah A, Barrera L, Gregoire F, Singh M, Chen D, Sabatino DE, Dong Y. Zhang X, et al. Among authors: barrera l. Sci Adv. 2020 Aug 21;6(34):eabc2315. doi: 10.1126/sciadv.abc2315. Print 2020 Aug. Sci Adv. 2020. PMID: 32937374 Free PMC article.
Cytosine base editing enables quadruple-edited allogeneic CART cells for T-ALL.
Diorio C, Murray R, Naniong M, Barrera L, Camblin A, Chukinas J, Coholan L, Edwards A, Fuller T, Gonzales C, Grupp SA, Ladd A, Le M, Messana A, Musenge F, Newman H, Poh YC, Poulin H, Ryan T, Shraim R, Tasian SK, Vincent T, Young L, Zhang Y, Ciaramella G, Gehrke J, Teachey DT. Diorio C, et al. Among authors: barrera l. Blood. 2022 Aug 11;140(6):619-629. doi: 10.1182/blood.2022015825. Blood. 2022. PMID: 35560156 Free PMC article.
Response to "Unexpected mutations after CRISPR-Cas9 editing in vivo".
Wilson CJ, Fennell T, Bothmer A, Maeder ML, Reyon D, Cotta-Ramusino C, Fernandez CA, Marco E, Barrera LA, Jayaram H, Albright CF, Cox GF, Church GM, Myer VE. Wilson CJ, et al. Among authors: barrera la. Nat Methods. 2018 Apr;15(4):236-237. doi: 10.1038/nmeth.4552. Epub 2018 Mar 30. Nat Methods. 2018. PMID: 29600989 No abstract available.
Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10.
Maeder ML, Stefanidakis M, Wilson CJ, Baral R, Barrera LA, Bounoutas GS, Bumcrot D, Chao H, Ciulla DM, DaSilva JA, Dass A, Dhanapal V, Fennell TJ, Friedland AE, Giannoukos G, Gloskowski SW, Glucksmann A, Gotta GM, Jayaram H, Haskett SJ, Hopkins B, Horng JE, Joshi S, Marco E, Mepani R, Reyon D, Ta T, Tabbaa DG, Samuelsson SJ, Shen S, Skor MN, Stetkiewicz P, Wang T, Yudkoff C, Myer VE, Albright CF, Jiang H. Maeder ML, et al. Among authors: barrera la. Nat Med. 2019 Feb;25(2):229-233. doi: 10.1038/s41591-018-0327-9. Epub 2019 Jan 21. Nat Med. 2019. PMID: 30664785
351 results