Recombinant virus products and methods for inhibition of expression of dux4
AssigneeThe Research Institute at Nationwide Children' s Hospital
InventorsScott Quenton Harper, Jian Liu, Sara Coppens, Lindsay Wallace
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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.
Claims18
1-12. (canceled)
A method of reducing DUX4-induced muscle degeneration or increasing muscle strength in a subject overexpressing DUX4 comprising administering to the subject an effective amount of a DNA comprising
- (a) a polynucleotide encoding miDUX4.405 miRNA comprising the nucleotide sequence of SEQ ID NO:
- 1;
- or
- (b) a polynucleotide encoding miDUX4.1156 miRNA comprising the nucleotide sequence of SEQ ID NO:
- 2.
A method of reducing DUX4-induced muscle degeneration or increasing muscle strength in a subject overexpressing DUX4 comprising administering to the subject an effective amount of a DNA encoding a DUX4 miRNA comprising a miRNA antisense guide strand, wherein the miRNA antisense guide strand comprises the nucleotide sequence of SEQ ID NO:
- 8482, SEQ ID NO:
- 8372, SEQ ID NO:
- 8371, SEQ ID NO:
- 8370, SEQ ID NO:
- 8367, SEQ ID NO:
- 8366, SEQ ID NO:
- 8365, SEQ ID NO:
- 8219, SEQ ID NO:
- 8218, SEQ ID NO:
- 8152, SEQ ID NO:
- 8147, SEQ ID NO:
- 8145, SEQ ID NO:
- 7397, SEQ ID NO:
- 7396, SEQ ID NO:
- 7395, SEQ ID NO:
- 7108, SEQ ID NO:
- 7107, SEQ ID NO:
- 7106, SEQ ID NO:
- 6633, SEQ ID NO:
- 6631, SEQ ID NO:
- 6622, SEQ ID NO:
- 6619, SEQ ID NO:
- 6609, SEQ ID NO:
- 6608, SEQ ID NO:
- 6568, SEQ ID NO:
- 6561 or SEQ ID NO:
- 6560.
Dependencies are inferred from the claim text.