Aav treatment of huntington's disease
AssigneeUniversity of Massachusetts
InventorsChristian Mueller, Neil Aronin, Edith L. Pfister
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Abstract
Aspects of the disclosure relate to compositions and methods useful for treating Huntington's disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.
Claims20
1-30. (canceled)
A cell comprising an isolated nucleic acid, wherein the isolated nucleic acid comprises a transgene encoding one or more mature, single-stranded miRNAs, wherein the nucleic acid sequence of the transgene encoding each mature, single-stranded miRNA comprises the sequence set forth in SEQ ID NO:
- 7, and is flanked by a heterologous miRNA backbone sequence.
A kit for producing a rAAV, comprising:
- (a) a container housing an isolated nucleic acid, wherein the isolated nucleic acid comprises a transgene encoding one or more mature, single-stranded miRNAs, wherein the nucleic acid sequence of the transgene encoding each mature, single-stranded miRNA comprises the sequence set forth in SEQ ID NO:
- 7, and is flanked by a heterologous miRNA backbone sequence;
- (b) a container housing an isolated nucleic acid encoding an AAV capsid protein;
- wherein the kit comprises instructions for production of the rAAV and use in administering the rAAV to a subject.
Dependencies are inferred from the claim text.