Intravenous delivery of an adenine base editor and a single-guide RNA for the Fah gene can correct an A>G splice-site mutation in an adult mouse mo......
Song, CQ; Jiang, T; Richter, M; Rhym, LH; Koblan, LW; Zafra, MP; Schatoff, EM; Doman, JL; Cao, YY; Dow, LE; Zhu, LJ; Anderson, DG; Liu, DR; Yin, H; Xue, W
Xue, W (corresponding author), Univ Massachusetts, Sch Med, RNA Therapeut Inst, Worcester, MA 01655 USA.; Liu, DR (corresponding author), Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA.; Liu, DR (corresponding author), Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA.; Liu, DR (corresponding author), Broad Inst MIT & Harvard, Cambridge, MA 02142 USA.; Xue, W (corresponding author), Univ Massachusetts, Sch Med, Dept Mol Cell & Canc Biol, Worcester, MA 01655 USA.; Xue, W (corresponding author), Univ Massachusetts, Sch Med, Dept Mol Med, Worcester, MA 01655 USA.; Yin, H (corresponding author), Wuhan Univ, Med Res Inst, Wuhan, Peoples R China.; Xue, W (corresponding author), Univ Massachusetts, Sch Med, Li Weibo Inst Rare Dis Res, Worcester, MA 01655 USA.
NATURE BIOMEDICAL ENGINEERING, 2020; 4 (1): 125