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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: michalakis s. Nat Commun. 2023 Oct 18;14(1):6578. doi: 10.1038/s41467-023-42386-0. Nat Commun. 2023. PMID: 37852949 Free PMC article.
In Vitro Evaluation of AAV Vectors for Retinal Gene Therapy.
Wagner JE, Schön C, Becirovic E, Biel M, Michalakis S. Wagner JE, et al. Among authors: michalakis s. Methods Mol Biol. 2019;1834:383-390. doi: 10.1007/978-1-4939-8669-9_24. Methods Mol Biol. 2019. PMID: 30324456
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. Böhm S, et al. Among authors: michalakis s. Sci Adv. 2020 Aug 19;6(34):eaba5614. doi: 10.1126/sciadv.aba5614. eCollection 2020 Aug. Sci Adv. 2020. PMID: 32875106 Free PMC article.
Gene therapy for achromatopsia.
Michalakis S, Schön C, Becirovic E, Biel M. Michalakis S, et al. J Gene Med. 2017 Mar;19(3). doi: 10.1002/jgm.2944. J Gene Med. 2017. PMID: 28095637 Review.
142 results