IP Library › Granted Patent US 11,446,396
Granted Patent B2
US 11,446,396 · App. 16/135,222 · Granted Sep 20, 2022

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.
A61K48/0058A61K38/1709A61K48/005A61K48/0066A61K48/0075A61M1/3603A61M5/1408C07K14/4707C12N7/00C12N2750/14143
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Quick Facts
Patent No.
US 11,446,396
App. No.
16/135,222
Granted
Sep 20, 2022
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 (6)

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 the patient with an encapsidated recombinant adeno-associated (AAV) comprising an AAVrh.74 capsid that comprises the amino acid sequence of SEQ ID NO: 2; wherein

the genome of the recombinant AAV comprises a gene expression cassette comprising the polynucleotide of SEQ ID NO: 3 encoding an alpha-sarcoglycan under the transcriptional control of a promoter, said cassette flanked by one or more AAV inverted terminal repeats.

2. The method of claim 1 , wherein the genome of the recombinant AAV is a self-complementary genome.

3. The method of claim 1 , wherein the promoter is tMCK.

4. The method of claim 1 , wherein the genome lacks AAV rep and cap DNA.

5. The method of claim 1 , wherein said patient is sequentially or subsequently administered a corticosteroid.

Assignments (2)
CONFIRMATORY LICENSE Recorded Feb 7, 2023
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 062668/0023 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Feb 16, 2021
From: MENDELL, JERRY R.; CHICOINE, LOUIS; RODINO-KLAPAC, LOUISE; CLARK, K. REED; PRESTON, THOMAS J.
To: NATIONWIDE CHILDREN'S HOSPITAL, INC.
Reel/Frame 055268/0637 →
Continuity (4)
Continuation 15255295 · Sep 2, 2016
Continuation 14360243
Provisional Application 61536139 · Nov 23, 2011
Related Publication 20190000998A1 · Jan 3, 2019