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:
Mar. 25, 2025
Filed:
Aug. 21, 2020
Applicant:
Research Institute AT Nationwide Children's Hospital, Columbus, OH (US);
Inventors:
Louise Rodino-Klapac, Grove City, OH (US);
Danielle Griffin, Canal Winchester, OH (US);
Jerry R. Mendell, Columbus, OH (US);
Assignee:
Research Institute at Nationwide Children's Hospital, Columbus, OH (US);
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/86 (2006.01); C07K 14/47 (2006.01); A61K 48/00 (2006.01); C07H 21/04 (2006.01);
U.S. Cl.
CPC ...
C12N 15/86 (2013.01); C07K 14/4716 (2013.01); A61K 48/005 (2013.01); C07H 21/04 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01);
Abstract
Described herein are methods of treating muscular dystrophy in a subject, comprising administration of a recombinant AAV vector AAVrh74.tMCK.hSGCA using a systemic route of administration and at a dose of about 1.0×10vg/kg to about 5.0×10vg/kg. Further disclosed are methods of expressing alpha-sarcoglycan gene in a cell or in a subject in need thereof, decreasing a serum CK level, and increasing alpha-sarcoglycan positive fibers in muscle tissue of a subject.