Compositions and methods for treating Huntington's disease
AssigneeVoyager Therapeutics, Inc.
InventorsDinah Wen-Yee Sah, Jinzhao Hou, Pengcheng Zhou, Xin Wang, Jochen Deckert, Markus Hossbach
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Abstract
The present invention relates to small interfering RNA (siRNA) molecules against the HTT gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating Huntington's Disease (HD) using the siRNA molecules and AAV vectors.
Claims29
A modulatory polynucleotide comprising the nucleotide sequence of SEQ ID NO:
- 349.
An adeno-associated viral (AAV) vector genome comprising a nucleic acid sequence positioned between two inverted terminal repeats (ITRs), wherein the nucleic acid sequence encodes a modulatory polynucleotide comprising the nucleotide sequence of SEQ ID NO:
- 349.
A method of inhibiting a huntingtin (HTT) gene, mRNA, and/or protein expression in a cell, comprising contacting the cell with an AAV vector genome comprising a nucleic acid sequence positioned between two inverted terminal repeats (ITRs), wherein the nucleic acid sequence encodes a modulatory polynucleotide comprising the nucleotide sequence of SEQ ID NO:
- 349,
- thereby inhibiting expression of the HTT gene, mRNA, and/or protein in the cell.
A method of treating Huntington's Disease (HD) in a subject, comprising administering to the subject an effective amount of an AAV vector genome comprising a nucleic acid sequence positioned between two inverted terminal repeats (ITRs), wherein the nucleic acid sequence encodes a modulatory polynucleotide comprising the nucleotide sequence of SEQ ID NO:
- 349,
- thereby treating HD in the subject.
Dependencies are inferred from the claim text.