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:
Oct. 31, 2023
Filed:
Mar. 12, 2019
Applicant:
Research Institute AT Nationwide Children's Hospital, Columbus, OH (US);
Inventors:
Scott Quenton Harper, Powell, OH (US);
Jian Liu, Los Angeles, CA (US);
Sara Coppens, Columbus, OH (US);
Lindsay Wallace, 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); C12N 15/113 (2010.01); C12N 15/09 (2006.01); C12N 15/63 (2006.01); C12N 15/11 (2006.01);
U.S. Cl.
CPC ...
C12N 15/86 (2013.01); C12N 15/113 (2013.01); C12N 2310/11 (2013.01); C12N 2310/141 (2013.01); C12N 2320/30 (2013.01); C12N 2330/51 (2013.01); C12N 2750/00032 (2013.01); C12N 2750/14132 (2013.01); C12N 2750/14143 (2013.01);
Abstract
The present invention relates to RNA interference-based methods for inhibiting the expression of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.