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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. Nat Commun. 2023 Oct 18;14(1):6578. doi: 10.1038/s41467-023-42386-0. Nat Commun. 2023. PMID: 37852949 Free PMC article.
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: riedmayr lm. Sci Adv. 2020 Aug 19;6(34):eaba5614. doi: 10.1126/sciadv.aba5614. eCollection 2020 Aug. Sci Adv. 2020. PMID: 32875106 Free PMC article.
In Vivo Analysis of Disease-Associated Point Mutations Unveils Profound Differences in mRNA Splicing of Peripherin-2 in Rod and Cone Photoreceptors.
Becirovic E, Böhm S, Nguyen ON, Riedmayr LM, Koch MA, Schulze E, Kohl S, Borsch O, Santos-Ferreira T, Ader M, Michalakis S, Biel M. Becirovic E, et al. Among authors: riedmayr lm. PLoS Genet. 2016 Jan 21;12(1):e1005811. doi: 10.1371/journal.pgen.1005811. eCollection 2016 Jan. PLoS Genet. 2016. PMID: 26796962 Free PMC article.
SMaRT for Therapeutic Purposes.
Riedmayr LM. Riedmayr LM. Methods Mol Biol. 2020;2079:219-232. doi: 10.1007/978-1-4939-9904-0_17. Methods Mol Biol. 2020. PMID: 31728974