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Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing.
Ding Q, Strong A, Patel KM, Ng SL, Gosis BS, Regan SN, Cowan CA, Rader DJ, Musunuru K. Ding Q, et al. Among authors: musunuru k. Circ Res. 2014 Aug 15;115(5):488-92. doi: 10.1161/CIRCRESAHA.115.304351. Epub 2014 Jun 10. Circ Res. 2014. PMID: 24916110 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.
Large, Diverse Population Cohorts of hiPSCs and Derived Hepatocyte-like Cells Reveal Functional Genetic Variation at Blood Lipid-Associated Loci.
Pashos EE, Park Y, Wang X, Raghavan A, Yang W, Abbey D, Peters DT, Arbelaez J, Hernandez M, Kuperwasser N, Li W, Lian Z, Liu Y, Lv W, Lytle-Gabbin SL, Marchadier DH, Rogov P, Shi J, Slovik KJ, Stylianou IM, Wang L, Yan R, Zhang X, Kathiresan S, Duncan SA, Mikkelsen TS, Morrisey EE, Rader DJ, Brown CD, Musunuru K. Pashos EE, et al. Among authors: musunuru k. Cell Stem Cell. 2017 Apr 6;20(4):558-570.e10. doi: 10.1016/j.stem.2017.03.017. Cell Stem Cell. 2017. PMID: 28388432 Free PMC article.
ESRP1 Mutations Cause Hearing Loss due to Defects in Alternative Splicing that Disrupt Cochlear Development.
Rohacek AM, Bebee TW, Tilton RK, Radens CM, McDermott-Roe C, Peart N, Kaur M, Zaykaner M, Cieply B, Musunuru K, Barash Y, Germiller JA, Krantz ID, Carstens RP, Epstein DJ. Rohacek AM, et al. Among authors: musunuru k. Dev Cell. 2017 Nov 6;43(3):318-331.e5. doi: 10.1016/j.devcel.2017.09.026. Epub 2017 Oct 26. Dev Cell. 2017. PMID: 29107558 Free PMC article.
232 results