inclusion of antisense-induced exon2 in alpha-glucosidase acid

摘要

The present disclosure relates to antisense oligomers and related compositions and methods for inducing the inclusion of exon as a treatment for type II glycogen storage disease (gsd-ii) (also known as pomp disease, glycogenosis ii, deficiency). (amd), acid alpha-glucosidase deficiency and lysosomal alpha-glucosidase deficiency), and more specifically refers to inducing the inclusion of exon 2 and thereby restoring enzymatically active acid alpha-glucosidase protein levels (gaa ) encoded by the gaa gene.