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State-of-the-art 2023 on gene therapy for phenylketonuria.
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.
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.
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.
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