申请人:Sarepta Therapeutics, Inc.
公开号:US10905709B2
公开(公告)日:2021-02-02
The present disclosure relates to modified antisense oligomers and related compositions and methods for increasing the expression of functional SMN protein and methods for treating spinal muscular atrophy and relates to inducing inclusion of exon 7 in SMN2 mRNA.
本公开涉及用于增加功能性 SMN 蛋白表达的修饰反义寡聚体及相关组合物和方法,以及用于治疗脊髓性肌萎缩症的方法,并涉及诱导 SMN2 mRNA 中外显子 7 的包含。