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:
Jun. 10, 2025
Filed:
Jul. 13, 2021
Applicant:
Nationwide Children's Hospital, Inc., Columbus, OH (US);
Inventors:
Scott Quenton Harper, Powell, OH (US);
Jian Liu, Savoy, IL (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); C12N 15/113 (2010.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); C07K 14/4707 (2013.01); C12N 15/1138 (2013.01); C12N 15/86 (2013.01); C12N 2310/14 (2013.01); C12N 2310/141 (2013.01); C12N 2320/32 (2013.01); C12N 2330/51 (2013.01); C12N 2750/14143 (2013.01);
Abstract
The present invention relates to RNA interference-based methods for inhibiting the expression of the myotilin gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of myotilin. The methods have application in the treatment of muscular dystrophies such as Limb Girdle Muscular Dystrophy Type 1A.