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Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo.
Yin H, Song CQ, Dorkin JR, Zhu LJ, Li Y, Wu Q, Park A, Yang J, Suresh S, Bizhanova A, Gupta A, Bolukbasi MF, Walsh S, Bogorad RL, Gao G, Weng Z, Dong Y, Koteliansky V, Wolfe SA, Langer R, Xue W, Anderson DG. Yin H, et al. Among authors: wolfe sa. Nat Biotechnol. 2016 Mar;34(3):328-33. doi: 10.1038/nbt.3471. Epub 2016 Feb 1. Nat Biotechnol. 2016. PMID: 26829318 Free PMC article.
Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing.
Yin H, Song CQ, Suresh S, Wu Q, Walsh S, Rhym LH, Mintzer E, Bolukbasi MF, Zhu LJ, Kauffman K, Mou H, Oberholzer A, Ding J, Kwan SY, Bogorad RL, Zatsepin T, Koteliansky V, Wolfe SA, Xue W, Langer R, Anderson DG. Yin H, et al. Among authors: wolfe sa. Nat Biotechnol. 2017 Dec;35(12):1179-1187. doi: 10.1038/nbt.4005. Epub 2017 Nov 13. Nat Biotechnol. 2017. PMID: 29131148 Free PMC article.
Partial DNA-guided Cas9 enables genome editing with reduced off-target activity.
Yin H, Song CQ, Suresh S, Kwan SY, Wu Q, Walsh S, Ding J, Bogorad RL, Zhu LJ, Wolfe SA, Koteliansky V, Xue W, Langer R, Anderson DG. Yin H, et al. Among authors: wolfe sa. Nat Chem Biol. 2018 Mar;14(3):311-316. doi: 10.1038/nchembio.2559. Epub 2018 Jan 29. Nat Chem Biol. 2018. PMID: 29377001 Free PMC article.
NmeCas9 is an intrinsically high-fidelity genome-editing platform.
Amrani N, Gao XD, Liu P, Edraki A, Mir A, Ibraheim R, Gupta A, Sasaki KE, Wu T, Donohoue PD, Settle AH, Lied AM, McGovern K, Fuller CK, Cameron P, Fazzio TG, Zhu LJ, Wolfe SA, Sontheimer EJ. Amrani N, et al. Among authors: wolfe sa. Genome Biol. 2018 Dec 5;19(1):214. doi: 10.1186/s13059-018-1591-1. Genome Biol. 2018. PMID: 30518407 Free PMC article.
Enhanced Cas12a editing in mammalian cells and zebrafish.
Liu P, Luk K, Shin M, Idrizi F, Kwok S, Roscoe B, Mintzer E, Suresh S, Morrison K, Frazão JB, Bolukbasi MF, Ponnienselvan K, Luban J, Zhu LJ, Lawson ND, Wolfe SA. Liu P, et al. Among authors: wolfe sa. Nucleic Acids Res. 2019 May 7;47(8):4169-4180. doi: 10.1093/nar/gkz184. Nucleic Acids Res. 2019. PMID: 30892626 Free PMC article.
Highly efficient therapeutic gene editing of human hematopoietic stem cells.
Wu Y, Zeng J, Roscoe BP, Liu P, Yao Q, Lazzarotto CR, Clement K, Cole MA, Luk K, Baricordi C, Shen AH, Ren C, Esrick EB, Manis JP, Dorfman DM, Williams DA, Biffi A, Brugnara C, Biasco L, Brendel C, Pinello L, Tsai SQ, Wolfe SA, Bauer DE. Wu Y, et al. Among authors: wolfe sa. Nat Med. 2019 May;25(5):776-783. doi: 10.1038/s41591-019-0401-y. Epub 2019 Mar 25. Nat Med. 2019. PMID: 30911135 Free PMC article.
342 results