In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model.
体内腺嘌呤碱基编辑可挽救人源化小鼠模型中的肾上腺脑白质营养不良症
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2024.05.027
Gopalappa Ramu, Lee MinYoung, Kim Globinna, Jung Eul Sik, Lee Hanahrae, Hwang Hye-Yeon, Lee Jong Geol, Kim Su Jung, Yoo Hyun Ju, Sung Young Hoon, Kim Daesik, Baek In-Jeoung, Kim Hyongbum Henry