A gene therapy for inherited blindness using dCas9-VPR-mediated transcriptional activation.
Böhm S, Splith V, Riedmayr LM, Rötzer RD, Gasparoni G, Nordström KJV, Wagner JE, Hinrichsmeyer KS, Walter J, Wahl-Schott C, Fenske S, Biel M, Michalakis S, Becirovic E.
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Sci Adv. 2020 Aug 19;6(34):eaba5614. doi: 10.1126/sciadv.aba5614. eCollection 2020 Aug.
Sci Adv. 2020.
PMID: 32875106
Free PMC article.