ANTISENSE-INDUCED INCLUSION OF EXON 2 IN AN ACID ALPHA-GLUCOSIDASE

Abstract

The present invention 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 pump disease, glycogenosis ii, maltase acid deficiency, glucosidase deficiency, and lysosomal alpha-glucosidase deficiency), and more specifically relates to the induction of exon 2 inclusion and thus the restoration of levels of the enzymatically active acid alpha-glucosidase (gaa) protein encoded by the gaa gene.