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74 results

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Page 1
Directed evolution of adenine base editors with increased activity and therapeutic application.
Gaudelli NM, Lam DK, Rees HA, Solá-Esteves NM, Barrera LA, Born DA, Edwards A, Gehrke JM, Lee SJ, Liquori AJ, Murray R, Packer MS, Rinaldi C, Slaymaker IM, Yen J, Young LE, Ciaramella G. Gaudelli NM, et al. Among authors: ciaramella g. Nat Biotechnol. 2020 Jul;38(7):892-900. doi: 10.1038/s41587-020-0491-6. Epub 2020 Apr 13. Nat Biotechnol. 2020. PMID: 32284586
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: ciaramella g. CRISPR J. 2021 Apr;4(2):169-177. doi: 10.1089/crispr.2020.0144. CRISPR J. 2021. PMID: 33876959
In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates.
Musunuru K, Chadwick AC, Mizoguchi T, Garcia SP, DeNizio JE, Reiss CW, Wang K, Iyer S, Dutta C, Clendaniel V, Amaonye M, Beach A, Berth K, Biswas S, Braun MC, Chen HM, Colace TV, Ganey JD, Gangopadhyay SA, Garrity R, Kasiewicz LN, Lavoie J, Madsen JA, Matsumoto Y, Mazzola AM, Nasrullah YS, Nneji J, Ren H, Sanjeev A, Shay M, Stahley MR, Fan SHY, Tam YK, Gaudelli NM, Ciaramella G, Stolz LE, Malyala P, Cheng CJ, Rajeev KG, Rohde E, Bellinger AM, Kathiresan S. Musunuru K, et al. Among authors: ciaramella g. Nature. 2021 May;593(7859):429-434. doi: 10.1038/s41586-021-03534-y. Epub 2021 May 19. Nature. 2021. PMID: 34012082
Evaluation of cytosine base editing and adenine base editing as a potential treatment for alpha-1 antitrypsin deficiency.
Packer MS, Chowdhary V, Lung G, Cheng LI, Aratyn-Schaus Y, Leboeuf D, Smith S, Shah A, Chen D, Zieger M, Cafferty BJ, Yan B, Ciaramella G, Gregoire FM, Mueller C. Packer MS, et al. Among authors: ciaramella g. Mol Ther. 2022 Apr 6;30(4):1396-1406. doi: 10.1016/j.ymthe.2022.01.040. Epub 2022 Feb 2. Mol Ther. 2022. PMID: 35121111 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: ciaramella g. Blood. 2022 Aug 11;140(6):619-629. doi: 10.1182/blood.2022015825. Blood. 2022. PMID: 35560156 Free PMC article.
Improved cytosine base editors generated from TadA variants.
Lam DK, Feliciano PR, Arif A, Bohnuud T, Fernandez TP, Gehrke JM, Grayson P, Lee KD, Ortega MA, Sawyer C, Schwaegerle ND, Peraro L, Young L, Lee SJ, Ciaramella G, Gaudelli NM. Lam DK, et al. Among authors: ciaramella g. Nat Biotechnol. 2023 May;41(5):686-697. doi: 10.1038/s41587-022-01611-9. Epub 2023 Jan 9. Nat Biotechnol. 2023. PMID: 36624149 Free PMC article.
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: ciaramella g. Nat Commun. 2024 Nov 10;15(1):9729. doi: 10.1038/s41467-024-54108-1. Nat Commun. 2024. PMID: 39523369 Free PMC article.
74 results