In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model.
Gopalappa R, Lee M, Kim G, Jung ES, Lee H, Hwang HY, Lee JG, Kim SJ, Yoo HJ, Sung YH, Kim D, Baek IJ, Kim HH.
Gopalappa R, et al. Among authors: sung yh.
Mol Ther. 2024 Jul 3;32(7):2190-2206. doi: 10.1016/j.ymthe.2024.05.027. Epub 2024 May 24.
Mol Ther. 2024.
PMID: 38796705