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CRISPR/Cas9 gene editing for curing sickle cell disease.
Park SH, Bao G. Park SH, et al. Transfus Apher Sci. 2021 Feb;60(1):103060. doi: 10.1016/j.transci.2021.103060. Epub 2021 Jan 10. Transfus Apher Sci. 2021. PMID: 33455878 Free PMC article. Review.
A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells.
Vakulskas CA, Dever DP, Rettig GR, Turk R, Jacobi AM, Collingwood MA, Bode NM, McNeill MS, Yan S, Camarena J, Lee CM, Park SH, Wiebking V, Bak RO, Gomez-Ospina N, Pavel-Dinu M, Sun W, Bao G, Porteus MH, Behlke MA. Vakulskas CA, et al. Among authors: park sh. Nat Med. 2018 Aug;24(8):1216-1224. doi: 10.1038/s41591-018-0137-0. Epub 2018 Aug 6. Nat Med. 2018. PMID: 30082871 Free PMC article.
LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study.
Doerfler AM, Park SH, Assini JM, Youssef A, Saxena L, Yaseen AB, De Giorgi M, Chuecos M, Hurley AE, Li A, Marcovina SM, Bao G, Boffa MB, Koschinsky ML, Lagor WR. Doerfler AM, et al. Among authors: park sh. Mol Ther Methods Clin Dev. 2022 Oct 13;27:337-351. doi: 10.1016/j.omtm.2022.10.009. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36381302 Free PMC article.
High levels of lipoprotein(a) in transgenic mice exacerbate atherosclerosis and promote vulnerable plaque features in a sex-specific manner.
Assini JM, Clark JR, Youssef A, Xing C, Doerfler AM, Park SH, Saxena L, Yaseen AB, Børen J, Gros R, Bao G, Lagor WR, Boffa MB, Koschinsky ML. Assini JM, et al. Among authors: park sh. Atherosclerosis. 2023 Nov;384:117150. doi: 10.1016/j.atherosclerosis.2023.05.019. Epub 2023 May 30. Atherosclerosis. 2023. PMID: 37290980
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