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Page 1
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: garcia sp. Nature. 2021 May;593(7859):429-434. doi: 10.1038/s41586-021-03534-y. Epub 2021 May 19. Nature. 2021. PMID: 34012082
In vivo CRISPR editing with no detectable genome-wide off-target mutations.
Akcakaya P, Bobbin ML, Guo JA, Malagon-Lopez J, Clement K, Garcia SP, Fellows MD, Porritt MJ, Firth MA, Carreras A, Baccega T, Seeliger F, Bjursell M, Tsai SQ, Nguyen NT, Nitsch R, Mayr LM, Pinello L, Bohlooly-Y M, Aryee MJ, Maresca M, Joung JK. Akcakaya P, et al. Among authors: garcia sp. Nature. 2018 Sep;561(7723):416-419. doi: 10.1038/s41586-018-0500-9. Epub 2018 Sep 12. Nature. 2018. PMID: 30209390 Free PMC article.
GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy.
Kasiewicz LN, Biswas S, Beach A, Ren H, Dutta C, Mazzola AM, Rohde E, Chadwick A, Cheng C, Garcia SP, Iyer S, Matsumoto Y, Khera AV, Musunuru K, Kathiresan S, Malyala P, Rajeev KG, Bellinger AM. Kasiewicz LN, et al. Among authors: garcia sp. Nat Commun. 2023 May 15;14(1):2776. doi: 10.1038/s41467-023-37465-1. Nat Commun. 2023. PMID: 37188660 Free PMC article.
BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis.
Canver MC, Smith EC, Sher F, Pinello L, Sanjana NE, Shalem O, Chen DD, Schupp PG, Vinjamur DS, Garcia SP, Luc S, Kurita R, Nakamura Y, Fujiwara Y, Maeda T, Yuan GC, Zhang F, Orkin SH, Bauer DE. Canver MC, et al. Among authors: garcia sp. Nature. 2015 Nov 12;527(7577):192-7. doi: 10.1038/nature15521. Epub 2015 Sep 16. Nature. 2015. PMID: 26375006 Free PMC article.
Molecularly distinct models of zebrafish Myc-induced B cell leukemia.
Borga C, Foster CA, Iyer S, Garcia SP, Langenau DM, Frazer JK. Borga C, et al. Among authors: garcia sp. Leukemia. 2019 Feb;33(2):559-562. doi: 10.1038/s41375-018-0328-1. Epub 2018 Dec 20. Leukemia. 2019. PMID: 30573774 Free PMC article. No abstract available.
66 results