The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Feb. 01, 2022
Filed:
Sep. 05, 2014
Sarepta Therapeutics, Inc., Cambridge, MA (US);
Murdoch University, Murdoch, AU;
Stephen Donald Wilton, Applecross, AU;
Sue Fletcher, Bayswater, AU;
Gunnar James Hanson, Cambridge, MA (US);
Richard Keith Bestwick, Corvallis, OR (US);
SAREPTA THERAPEUTICS, INC., Cambridge, MA (US);
MURDOCH UNIVERSITY, Murdoch, AU;
Abstract
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.