WILTON, STEPHEN DONALD,FLETCHER, SUE,HANSON, GUNNAR JAMES,BESTWICK, RICHARD KEITH,SCHNELL, FREDERICK J.
申请号：
CA2977528
公开号：
CA2977528A1
申请日:
2016.02.29
申请国别(地区):
CA
年份:
2016
代理人:
摘要：
The present disclosure relates to antisense oligomers and related compositions and methods for inducing exon inclusion as a treatment for glycogen storage disease type II (GSD- II) (also known as Pompe disease, glycogenosis II, acid maltase deficiency (AMD), acid alpha- glucosidase deficiency, and lysosomal alpha-glucosidase deficiency), and more specifically relates to inducing inclusion of exon 2 and thereby restoring levels of enzymatically active acid alpha-glucosidase (GAA) protein encoded by the GAA gene.
