| abstract |
PROBLEM TO BE SOLVED: To provide antisense-induced exon 2 inclusion in acid α-glucosidase. The present disclosure relates to type II glycogen storage disorder (GSD-II) (Pompe disease, glycogen storage disease II, acid maltase deficiency (AMD), acid α-glucosidase deficiency, and lysosomal α-glucosidase deficiency. (Also known as symptom) and antisense oligomers and related compositions and methods for the induction of exon inclusion, and more specifically, induces exon 2 inclusion, thereby providing a recovery level of the GAA gene. To obtain an enzymatically active acid α-glucosidase (GAA) protein encoded by [Selection diagram] None |