Weber VJ, Reschigna A, Gerhardt MJ, Heigl T, Hinrichsmeyer KS, van den Engel S, Otify DY, Gavrilov Z, Blaser F, Meneau I, Betz C, Bolz HJ, Biel M, Michalakis S, Becirovic E. CRISPR/Cas-mediated activation of genes associated with inherited retinal dystrophies in human cells for diagnostic purposes. JCI Insight. 2025 Sep 30:e189615. doi: 10.1172/jci.insight.189615. Epub ahead of print. PMID: 41056201.

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. mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy. Nat Commun. 2023 Oct 18;14(1):6578. doi: 10.1038/s41467-023-42386-0. PMID: 37852949; PMCID: PMC10584818.

Occelli LM, Zobel L, Stoddard J, Wagner J, Pasmanter N, Querubin J, Renner LM, Reynaga R, Winkler PA, Sun K, Marinho LFLP, O'Riordan CR, Frederick A, Lauer A, Tsang SH, Hauswirth WW, McGill TJ, Neuringer M, Michalakis S, Petersen-Jones SM. Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa. Mol Ther. 2023 Jul 5;31(7):2028-2041. doi: 10.1016/j.ymthe.2023.04.005. Epub 2023 Apr 13. PMID: 37056049; PMCID: PMC10362398.

Kandaswamy S, Zobel L, John B, Santhiya ST, Bogedein J, Przemeck GKH, Gailus-Durner V, Fuchs H, Biel M, de Angelis MH, Graw J, Michalakis S, Amarie OV. Mutations within the cGMP-binding domain of CNGA1 causing autosomal recessive retinitis pigmentosa in human and animal model. Cell Death Discov. 2022 Sep 17;8(1):387. doi: 10.1038/s41420-022-01185-0. PMID: 36115851; PMCID: PMC9482621.

Riedmayr LM, Hinrichsmeyer KS, Karguth N, Böhm S, Splith V, Michalakis S, Becirovic E. dCas9-VPR-mediated transcriptional activation of functionally equivalent genes for gene therapy. Nat Protoc. 2022 Mar;17(3):781-818. doi: 10.1038/s41596-021-00666-3. Epub 2022 Feb 7. PMID: 35132255.

Traube FR, Özdemir D, Sahin H, Scheel C, Glück AF, Geserich AS, Oganesian S, Kostidis S, Iwan K, Rahimoff R, Giorgio G, Müller M, Spada F, Biel M, Cox J, Giera M, Michalakis S, Carell T. Redirected nuclear glutamate dehydrogenase supplies Tet3 with α-ketoglutarate in neurons. Nat Commun. 2021 Jul 2;12(1):4100. doi: 10.1038/s41467-021-24353-9. PMID: 34215750; PMCID: PMC8253819.

Völkner M, Pavlou M, Büning H, Michalakis S, Karl MO. Optimized Adeno-Associated Virus Vectors for Efficient Transduction of Human Retinal Organoids. Hum Gene Ther. 2021 Jul;32(13-14):694-706. doi: 10.1089/hum.2020.321. Epub 2021 Jun 29. PMID: 33752467.

Pavlou M, Schön C, Occelli LM, Rossi A, Meumann N, Boyd RF, Bartoe JT, Siedlecki J, Gerhardt MJ, Babutzka S, Bogedein J, Wagner JE, Priglinger SG, Biel M, Petersen-Jones SM, Büning H, Michalakis S. Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders. EMBO Mol Med. 2021 Apr 9;13(4):e13392. doi: 10.15252/emmm.202013392. Epub 2021 Feb 22. PMID: 33616280; PMCID: PMC8033523.

Murenu E, Pavlou M, Richter L, Rapti K, Just S, Cehajic-Kapetanovic J, Tafrishi N, Hayes A, Scholey R, Lucas R, Büning H, Grimm D, Michalakis S. A universal protocol for isolating retinal ON bipolar cells across species via fluorescence-activated cell sorting. Mol Ther Methods Clin Dev. 2021 Jan 26;20:587-600. doi: 10.1016/j.omtm.2021.01.011. PMID: 33665228; PMCID: PMC7895692.

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. 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. PMID: 32875106; PMCID: PMC7438099.