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2016 2
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2019 3
2020 2
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ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome.
Ou L, DeKelver RC, Rohde M, Tom S, Radeke R, St Martin SJ, Santiago Y, Sproul S, Przybilla MJ, Koniar BL, Podetz-Pedersen KM, Laoharawee K, Cooksley RD, Meyer KE, Holmes MC, McIvor RS, Wechsler T, Whitley CB. Ou L, et al. Among authors: przybilla mj. Mol Ther. 2019 Jan 2;27(1):178-187. doi: 10.1016/j.ymthe.2018.10.018. Epub 2018 Nov 1. Mol Ther. 2019. PMID: 30528089 Free PMC article.
A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases.
Ou L, Przybilla MJ, Tăbăran AF, Overn P, O'Sullivan MG, Jiang X, Sidhu R, Kell PJ, Ory DS, Whitley CB. Ou L, et al. Among authors: przybilla mj. Gene Ther. 2020 May;27(5):226-236. doi: 10.1038/s41434-019-0120-5. Epub 2020 Jan 2. Gene Ther. 2020. PMID: 31896760 Free PMC article.
11 results