In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression
体内腺嘌呤碱基编辑突变型Galc基因可改善克拉伯病进展
期刊:Genome Medicine
影响因子:11.2
doi:10.1186/s13073-026-01620-2
Nam, Bae-Geun; Seo, Jung Hwa; Hong, Sung-Ah; Kim, Ju-Hee; Kang, Minju; Notario, Geneva Rose; Hong, Yoontaik; Cho, Seunghee; Bae, Sangsu; Cho, Sung-Rae