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Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10.
Maeder ML, Stefanidakis M, Wilson CJ, Baral R, Barrera LA, Bounoutas GS, Bumcrot D, Chao H, Ciulla DM, DaSilva JA, Dass A, Dhanapal V, Fennell TJ, Friedland AE, Giannoukos G, Gloskowski SW, Glucksmann A, Gotta GM, Jayaram H, Haskett SJ, Hopkins B, Horng JE, Joshi S, Marco E, Mepani R, Reyon D, Ta T, Tabbaa DG, Samuelsson SJ, Shen S, Skor MN, Stetkiewicz P, Wang T, Yudkoff C, Myer VE, Albright CF, Jiang H. Maeder ML, et al. Among authors: jiang h. Nat Med. 2019 Feb;25(2):229-233. doi: 10.1038/s41591-018-0327-9. Epub 2019 Jan 21. Nat Med. 2019. PMID: 30664785
Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque.
McCullough KT, Boye SL, Fajardo D, Calabro K, Peterson JJ, Strang CE, Chakraborty D, Gloskowski S, Haskett S, Samuelsson S, Jiang H, Witherspoon CD, Gamlin PD, Maeder ML, Boye SE. McCullough KT, et al. Among authors: jiang h. Hum Gene Ther. 2019 May;30(5):571-589. doi: 10.1089/hum.2018.193. Epub 2018 Dec 20. Hum Gene Ther. 2019. PMID: 30358434 Free PMC article.
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