Recombinant virus products and methods for inhibition of expression of DUX4
AssigneeNationwide Children's Hospital, Inc.
InventorsScott Quenton Harper, Jian Liu, Sara Garwick Coppens, Lindsay Wallace
Life of this patent
Estimating the term…
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.
Claims5
A recombinant adeno-associated virus comprising the nucleotide sequence of SEQ ID NO:
- 1 or SEQ ID NO:
- 2 encoding a DUX4 miRNA, wherein the recombinant adeno-associated virus lacks rep and cap genes.
A method of treating a facioscapulohumeral muscular dystrophy patient comprising administering to the patient by intramuscular injection a recombinant adeno-associated virus comprising nucleotide sequence of SEQ ID NO:
- 1 encoding a DUX4 miRNA or the nucleotide sequence of SEQ ID NO:
- 2 encoding a DUX4 miRNA, wherein the recombinant adeno-associated virus lacks rep and cap genes.
The nucleotide sequence of SEQ ID NO:
- 1 encoding miDUX4.405 or the nucleotide sequence of SEQ ID NO:
- 2 encoding miDUX4.1156.
Dependencies are inferred from the claim text.