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A gene therapy for inherited blindness using dCas9-VPR-mediated transcriptional activation.
Sci Adv. 2020 Aug 19;6(34):eaba5614. doi: 10.1126/sciadv.aba5614. eCollection 2020 Aug.
Sci Adv. 2020.
PMID: 32875106
Free PMC article.
dCas9-VPR-mediated transcriptional activation of functionally equivalent genes for gene therapy.
Riedmayr LM, Hinrichsmeyer KS, Karguth N, Böhm S, Splith V, Michalakis S, Becirovic E.
Riedmayr LM, et al. Among authors: hinrichsmeyer ks.
Nat Protoc. 2022 Mar;17(3):781-818. doi: 10.1038/s41596-021-00666-3. Epub 2022 Feb 7.
Nat Protoc. 2022.
PMID: 35132255
Review.
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mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy.
Riedmayr LM, Hinrichsmeyer KS, Thalhammer SB, Mittas DM, Karguth N, Otify DY, Böhm S, Weber VJ, Bartoschek MD, Splith V, Brümmer M, Ferreira R, Boon N, Wögenstein GM, Grimm C, Wijnholds J, Mehlfeld V, Michalakis S, Fenske S, Biel M, Becirovic E.
Riedmayr LM, et al. Among authors: hinrichsmeyer ks.
Nat Commun. 2023 Oct 18;14(1):6578. doi: 10.1038/s41467-023-42386-0.
Nat Commun. 2023.
PMID: 37852949
Free PMC article.
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