EP-4488370-A3 - RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSSION OF DUX4
EP4488370A3EP 4488370 A3EP4488370 A3EP 4488370A3EP-4488370-A3
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.
Inventors
- HARPER, Scott Quenton
- LIU, JIAN
- GARWICK, Sara
- WALLACE, Lindsay
Assignees
- Nationwide Children's Hospital, Inc.
Dates
- Publication Date
- 20260506
- Application Date
- 20120724