Modulating splicing in 5' untranslated regions to treat rare haploinsufficient disease
通过调控5'非翻译区的剪接来治疗罕见的单倍体不足疾病
期刊:
影响因子:
doi:10.64898/2025.12.07.692584
Beer Wells, Eloise S; De Conti, Laura; Kim, Hyung Chul; Rohani, Narjes; Chundru, Kartik; Watts, Laura M; Dawes, Ruebena; Chen, Yuyang; Martin-Geary, Alexandra C; Griffiths, Michael J; Scott, Sam; Bamford, Rosemary A; Mill, Jonathan; Wright, Caroline F; Baralle, Marco; Sanders, Stephan J; Whiffin, Nicola