Enhanced HDR-mediated correction of heterozygous COL7A1 mutations for recessive dystrophic epidermolysis bullosa
增强 HDR 介导的隐性营养不良性大疱性表皮松解症杂合 COL7A1 突变的校正
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102472
Hunt, John M T; du Rand, Alex; Verdon, Daniel; Clemance, Leah; Loef, Evert; Malhi, Chloe; Buttle, Ben; Knapp, David J H F; Michaels, Yale S; Garlick, Jonathan; Dunbar, P Rod; Purvis, Diana; Feisst, Vaughan; Sheppard, Hilary