Haplotype editing with CRISPR-Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFL
利用 CRISPR-Cas9 进行单倍型编辑作为治疗 NEFL 中显性负性错义突变的治疗方法
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.11.026
Dua, Poorvi H; Simon, Bazilco M J; Marley, Chiara B E; Feliciano, Carissa M; Watry, Hannah L; Cowan, Quinn T; Steury, Dylan; Abraham, Abin; Gilbertson, Erin N; Ramey, Grace D; Capra, John A; Conklin, Bruce R; Judge, Luke M