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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: hruzova m. 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 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: hruzova m. 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.
Palmitoylation of BMPR1a regulates neural stem cell fate.
Wegleiter T, Buthey K, Gonzalez-Bohorquez D, Hruzova M, Bin Imtiaz MK, Abegg A, Mebert I, Molteni A, Kollegger D, Pelczar P, Jessberger S. Wegleiter T, et al. Among authors: hruzova m. Proc Natl Acad Sci U S A. 2019 Dec 17;116(51):25688-25696. doi: 10.1073/pnas.1912671116. Epub 2019 Nov 26. Proc Natl Acad Sci U S A. 2019. PMID: 31772009 Free PMC article.