Self-induced correction of the genetic defect in tyrosinemia type I - PubMed (original) (raw)

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. 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.