Methods for treating huntington's disease
AssigneesAsklepios Biopharmaceutical, Inc., The University of North Carolina at Chapel Hill
InventorsSiewHui LOW, Xiao XIAO, Philippe MOULLIER, Bin XIAO
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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. Accordingly, in some aspects, the disclosure provides an isolated nucleic acid comprising or encoding the sequence set forth in any one of SEQ ID NOs: 1-22.
Claims26
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An isolated nucleic acid comprising:
- (a) a first region comprising a first adeno-associated virus (AAV) inverted terminal repeat (ITR), or a variant thereof;
- and
- (b) a second region comprising a transgene encoding one or more miRNAs, wherein each miRNA is encoded by a sequence comprising the sequence set forth in any one of SEQ ID NOs:
- 1-22 flanked by a miRNA backbone sequence.
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Dependencies are inferred from the claim text.