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Efficient prime editing in mouse brain, liver and heart with dual AAVs.
Davis JR, Banskota S, Levy JM, Newby GA, Wang X, Anzalone AV, Nelson AT, Chen PJ, Hennes AD, An M, Roh H, Randolph PB, Musunuru K, Liu DR. Davis JR, et al. Among authors: musunuru k. Nat Biotechnol. 2024 Feb;42(2):253-264. doi: 10.1038/s41587-023-01758-z. Epub 2023 May 4. Nat Biotechnol. 2024. PMID: 37142705 Free PMC article.
CRISPR-Cas9 Targeting of PCSK9 in Human Hepatocytes In Vivo-Brief Report.
Wang X, Raghavan A, Chen T, Qiao L, Zhang Y, Ding Q, Musunuru K. Wang X, et al. Among authors: musunuru k. Arterioscler Thromb Vasc Biol. 2016 May;36(5):783-6. doi: 10.1161/ATVBAHA.116.307227. Epub 2016 Mar 3. Arterioscler Thromb Vasc Biol. 2016. PMID: 26941020 Free PMC article.
Angiopoietin-Like 3: From Discovery to Therapeutic Gene Editing.
Wang X, Musunuru K. Wang X, et al. Among authors: musunuru k. JACC Basic Transl Sci. 2019 Oct 28;4(6):755-762. doi: 10.1016/j.jacbts.2019.05.008. eCollection 2019 Oct. JACC Basic Transl Sci. 2019. PMID: 31709322 Free PMC article. Review.
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. Nature. 2021 May;593(7859):429-434. doi: 10.1038/s41586-021-03534-y. Epub 2021 May 19. Nature. 2021. PMID: 34012082
Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins.
Banskota S, Raguram A, Suh S, Du SW, Davis JR, Choi EH, Wang X, Nielsen SC, Newby GA, Randolph PB, Osborn MJ, Musunuru K, Palczewski K, Liu DR. Banskota S, et al. Among authors: musunuru k. Cell. 2022 Jan 20;185(2):250-265.e16. doi: 10.1016/j.cell.2021.12.021. Epub 2022 Jan 11. Cell. 2022. PMID: 35021064 Free PMC article.
226 results