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183 results

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Page 1
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.
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.
A liver-humanized mouse model of carbamoyl phosphate synthetase 1-deficiency.
Srinivasan RC, Zabulica M, Hammarstedt C, Wu T, Gramignoli R, Kannisto K, Ellis E, Karadagi A, Fingerhut R, Allegri G, Rüfenacht V, Thöny B, Häberle J, Nuoffer JM, Strom SC. Srinivasan RC, et al. Among authors: thony b. J Inherit Metab Dis. 2019 Nov;42(6):1054-1063. doi: 10.1002/jimd.12067. Epub 2019 Apr 2. J Inherit Metab Dis. 2019. PMID: 30843237 Free article.
Analysis of the Qatari R336C cystathionine β-synthase protein in mice.
Gupta S, Gallego-Villar L, Wang L, Lee HO, Nasrallah G, Al-Dewik N, Häberle J, Thöny B, Blom HJ, Ben-Omran T, Kruger WD. Gupta S, et al. Among authors: thony b. J Inherit Metab Dis. 2019 Sep;42(5):831-838. doi: 10.1002/jimd.12140. Epub 2019 Jul 10. J Inherit Metab Dis. 2019. PMID: 31240737 Free PMC 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.
Editorial.
Häberle J, Thöny B. Häberle J, et al. Among authors: thony b. J Inherit Metab Dis. 2019 Nov;42(6):1041-1043. doi: 10.1002/jimd.12179. J Inherit Metab Dis. 2019. PMID: 31769067 Free article. No abstract available.
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.
183 results