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Page 1
Therapeutic liver cell transplantation to treat murine PKU.
Willimann M, Grisch-Chan HM, Rimann N, Rothgangl T, Hruzova M, Schwank G, Thöny B. Willimann M, et al. Among authors: rothgangl t. J Inherit Metab Dis. 2024 Nov;47(6):1322-1335. doi: 10.1002/jimd.12802. Epub 2024 Oct 24. J Inherit Metab Dis. 2024. PMID: 39449255 Free PMC article.
Effective genome editing with an enhanced ISDra2 TnpB system and deep learning-predicted ωRNAs.
Marquart KF, Mathis N, Mollaysa A, Müller S, Kissling L, Rothgangl T, Schmidheini L, Kulcsár PI, Allam A, Kaufmann MM, Matsushita M, Haenggi T, Cathomen T, Kopf M, Krauthammer M, Schwank G. Marquart KF, et al. Among authors: rothgangl t. Nat Methods. 2024 Nov;21(11):2084-2093. doi: 10.1038/s41592-024-02418-z. Epub 2024 Sep 23. Nat Methods. 2024. PMID: 39313558
Enhancing prime editor activity by directed protein evolution in yeast.
Weber Y, Böck D, Ivașcu A, Mathis N, Rothgangl T, Ioannidi EI, Blaudt AC, Tidecks L, Vadovics M, Muramatsu H, Reichmuth A, Marquart KF, Kissling L, Pardi N, Jinek M, Schwank G. Weber Y, et al. Among authors: rothgangl t. Nat Commun. 2024 Mar 7;15(1):2092. doi: 10.1038/s41467-024-46107-z. Nat Commun. 2024. PMID: 38453904 Free PMC article.
In vivo targeting of a variant causing vanishing white matter using CRISPR/Cas9.
Hillen AEJ, Hruzova M, Rothgangl T, Breur M, Bugiani M, van der Knaap MS, Schwank G, Heine VM. Hillen AEJ, et al. Among authors: rothgangl t. Mol Ther Methods Clin Dev. 2022 Feb 23;25:17-25. doi: 10.1016/j.omtm.2022.02.006. eCollection 2022 Jun 9. Mol Ther Methods Clin Dev. 2022. PMID: 35317047 Free PMC article.
In vivo prime editing of a metabolic liver disease in mice.
Böck D, Rothgangl T, Villiger L, Schmidheini L, Matsushita M, Mathis N, Ioannidi E, Rimann N, Grisch-Chan HM, Kreutzer S, Kontarakis Z, Kopf M, Thöny B, Schwank G. Böck D, et al. Among authors: rothgangl t. Sci Transl Med. 2022 Mar 16;14(636):eabl9238. doi: 10.1126/scitranslmed.abl9238. Epub 2022 Mar 16. Sci Transl Med. 2022. PMID: 35294257 Free PMC article.
In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels.
Rothgangl T, Dennis MK, Lin PJC, Oka R, Witzigmann D, Villiger L, Qi W, Hruzova M, Kissling L, Lenggenhager D, Borrelli C, Egli S, Frey N, Bakker N, Walker JA 2nd, Kadina AP, Victorov DV, Pacesa M, Kreutzer S, Kontarakis Z, Moor A, Jinek M, Weissman D, Stoffel M, van Boxtel R, Holden K, Pardi N, Thöny B, Häberle J, Tam YK, Semple SC, Schwank G. Rothgangl T, et al. Nat Biotechnol. 2021 Aug;39(8):949-957. doi: 10.1038/s41587-021-00933-4. Epub 2021 May 19. Nat Biotechnol. 2021. PMID: 34012094 Free PMC article.
In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA.
Villiger L, Rothgangl T, Witzigmann D, Oka R, Lin PJC, Qi W, Janjuha S, Berk C, Ringnalda F, Beattie MB, Stoffel M, Thöny B, Hall J, Rehrauer H, van Boxtel R, Tam YK, Schwank G. Villiger L, et al. Among authors: rothgangl t. Nat Biomed Eng. 2021 Feb;5(2):179-189. doi: 10.1038/s41551-020-00671-z. Epub 2021 Jan 25. Nat Biomed Eng. 2021. PMID: 33495639 Free PMC article.