IP Library › Granted Patent US 10,105,453
Granted Patent B2
US 10,105,453 · App. 15/255,295 · Granted Oct 23, 2018

Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides

Inventors: Jerry R. Mendell (Columbus, OH); Louis Chicoine (Westerville, OH); Louise Rodino-Klapac (Groveport, OH); Kelly Reed Clark (Columbus, OH); Thomas J. Preston (Groveport, OH)
Assignee: NATIONWIDE CHILDREN'S HOSPITAL, INC.
A61K48/0058A61K38/1709A61K48/0066A61K48/0075C07K14/4707C12N7/00C12N2750/14143
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Quick Facts
Patent No.
US 10,105,453
App. No.
15/255,295
Granted
Oct 23, 2018
Kind
B2
Abstract

The present invention relates to recombinant adeno-associated virus (rAAV) delivery of an alpha-sarcoglycan gene. The invention provides rAAV products and methods of using the rAAV in the treatment of limb girdle muscular dystrophies such as LGMD2D.

Claims (1)

1. A method of improving muscle function in a patient afflicted with limb girdle muscular dystrophy type 2D (LGMD 2D) comprising the step of administering by vascular delivery to the patient a recombinant adeno-associated virus (AAV) comprising an AAV rh.74 capsid of SEQ ID NO: 2, wherein the genome of the recombinant AAV is a self-complementary genome which comprises a gene expression cassette comprising the alpha-sarcoglycan polynucleotide of SEQ ID NO: 3 under the transcriptional control of a promoter, said cassette flanked by one or more AAV inverted terminal repeats, and wherein the recombinant AAV genome lacks AAV rep and cap DNA.

Assignments (2)
CONFIRMATORY LICENSE Recorded Feb 28, 2017
From: CHILDREN'S HOSPITAL (COLUMBUS)
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 041825/0564 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Nov 15, 2016
From: MENDELL, JERRY; CHICOINE, LOUIS; RODINO-KLAPAC, LOUISE; CLARK, K. REED; PRESTON, THOMAS J.
To: NATIONWIDE CHILDREN'S HOSPITAL, INC.
Reel/Frame 040326/0241 →
Continuity (3)
Continuation 14360243
Provisional Application 61563139 · Nov 23, 2011
Related Publication 20170209598A1 · Jul 27, 2017
Cited By (7)
US 12,258,573 US 12,263,230 US 12,285,497 US 12,377,170 US 12,391,928 US 12,472,239 US 12,735,723