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: kim hh.
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Mol Ther. 2024.
PMID: 38796705