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Targeted knock-in of NCF1 cDNA into the NCF2 locus leads to myeloid phenotypic correction of p47 phox -deficient chronic granulomatous disease.
Siow KM, Güngör M, Wrona D, Raimondi F, Pastukhov O, Tsapogas P, Menzi T, Schmitz M, Kulcsár PI, Schwank G, Schulz A, Jinek M, Modlich U, Siler U, Reichenbach J. Siow KM, et al. Among authors: schwank g. Mol Ther Nucleic Acids. 2024 May 24;35(3):102229. doi: 10.1016/j.omtn.2024.102229. eCollection 2024 Sep 10. Mol Ther Nucleic Acids. 2024. PMID: 38952440 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: schwank g. 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 Free PMC article. Review.
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: schwank g. 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.
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: schwank g. 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.
Genetic ablation of ketohexokinase C isoform impairs pancreatic cancer development.
Guccini I, Tang G, To TT, Di Rito L, Le Blanc S, Strobel O, D'Ambrosio M, Pasquini E, Bolis M, Silva P, Kabakci HA, Godbersen S, Alimonti A, Schwank G, Stoffel M. Guccini I, et al. Among authors: schwank g. iScience. 2023 Jul 13;26(8):107368. doi: 10.1016/j.isci.2023.107368. eCollection 2023 Aug 18. iScience. 2023. PMID: 37559908 Free PMC article.
51 results