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183 results

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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: thony b. 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.
Treatment of a metabolic liver disease by in vivo genome base editing in adult mice.
Villiger L, Grisch-Chan HM, Lindsay H, Ringnalda F, Pogliano CB, Allegri G, Fingerhut R, Häberle J, Matos J, Robinson MD, Thöny B, Schwank G. Villiger L, et al. Among authors: thony b. Nat Med. 2018 Oct;24(10):1519-1525. doi: 10.1038/s41591-018-0209-1. Epub 2018 Oct 8. Nat Med. 2018. PMID: 30297904 Free article.
State-of-the-Art 2019 on Gene Therapy for Phenylketonuria.
Grisch-Chan HM, Schwank G, Harding CO, Thöny B. Grisch-Chan HM, et al. Among authors: thony b. Hum Gene Ther. 2019 Oct;30(10):1274-1283. doi: 10.1089/hum.2019.111. Epub 2019 Sep 9. Hum Gene Ther. 2019. PMID: 31364419 Free PMC article. Review.
Correction of a urea cycle defect after ex vivo gene editing of human hepatocytes.
Zabulica M, Srinivasan RC, Akcakaya P, Allegri G, Bestas B, Firth M, Hammarstedt C, Jakobsson T, Jakobsson T, Ellis E, Jorns C, Makris G, Scherer T, Rimann N, van Zuydam NR, Gramignoli R, Forslöw A, Engberg S, Maresca M, Rooyackers O, Thöny B, Häberle J, Rosen B, Strom SC. Zabulica M, et al. Among authors: thony b. Mol Ther. 2021 May 5;29(5):1903-1917. doi: 10.1016/j.ymthe.2021.01.024. Epub 2021 Jan 21. Mol Ther. 2021. PMID: 33484963 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. Among authors: thony b. 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 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: thony b. 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.
State-of-the-art 2023 on gene therapy for phenylketonuria.
Martinez M, Harding CO, Schwank G, Thöny B. Martinez M, et al. Among authors: thony b. J Inherit Metab Dis. 2024 Jan;47(1):80-92. doi: 10.1002/jimd.12651. Epub 2023 Aug 3. J Inherit Metab Dis. 2024. PMID: 37401651 Review.
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: thony b. J Inherit Metab Dis. 2024 Oct 24. doi: 10.1002/jimd.12802. Online ahead of print. J Inherit Metab Dis. 2024. PMID: 39449255
183 results