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US 2023/0399642 A1Published application
Compositions and methods of treating huntington's disease
AssigneeVoyager Therapeutics, Inc.
InventorsJinzhao Hou, Xin Wang, Dinah Wen-Yee Sah, Pengcheng Zhou
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Abstract
The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).
Claims19
An adeno-associated virus (AAV) viral genome, comprising:
- (a) a 5′ inverted terminal repeat (ITR) sequence region, wherein said 5′ ITR sequence region comprises SEQ ID NO:
- 50 or 52;
- (b) an enhancer sequence region, wherein said enhancer sequence region comprises SEQ ID NO:
- 54 or 55;
- (c) a promoter sequence region, wherein said promoter sequence region comprises SEQ ID NO:
- 56, 57 or 58;
- (d) a modulatory polynucleotide sequence region, wherein said modulatory polynucleotide sequence region comprises any one of SEQ ID NO:
- 23, 24, 25, 26, 27, or 28;
- (e) a polyadenylation (polyA) signal sequence region, wherein said polyA signal sequence region comprises SEQ ID NO:
- 66 or 67;
- and
- (f) a 3′ ITR sequence region, wherein said 3′ ITR sequence region comprises SEQ ID NO:
- 51 or 53.
10 more elements
An adeno-associated virus (AAV) viral genome comprising the nucleotide sequence of any one of SEQ ID NOs:
- 39-44, or a nucleotide sequence at least 95% identical thereto.
-16. (canceled)
-25. (canceled)
Dependencies are inferred from the claim text.