Gene Therapy via Trans-Splicing for LMNA-Related Congenital Muscular Dystrophy
通过反式剪接进行 LMNA 相关先天性肌营养不良症的基因治疗
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.5
doi:10.1016/j.omtn.2017.12.012
Feriel Azibani, Astrid Brull, Ludovic Arandel, Maud Beuvin, Isabelle Nelson, Arnaud Jollet, Esma Ziat, Bernard Prudhon, Sofia Benkhelifa-Ziyyat, Marc Bitoun, Stéphanie Lorain, Gisèle Bonne, Anne T Bertrand