Compositions for treating Friedreich's ataxia
AssigneeThe Trustees of the University of Pennsylvania
InventorsJames M. Wilson, Christian Hinderer, Nimrod Miller
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Abstract
A recombinant adeno-associated virus (rAAV) comprising an AAV capsid and a vector genome comprising a frataxin gene is provided. Also provided is a composition containing an effective amount of rAAV to ameliorate symptoms of Freidreich's ataxia, including, e.g., reduction in progression towards neurocognitive decline and/or cardiomyopathy.
Claims22
A recombinant adeno-associated virus (rAAV) having an AAV capsid and a vector genome comprising a FXN gene having the sequence of SEQ ID NO:
- 3 or a sequence 95% identical thereto that encodes human frataxin protein having amino acid sequence of SEQ ID NO:
- 2, and regulatory sequences which direct expression of the FXN gene in targeted human cells.
A plasmid comprising an expression cassette comprising a FXN gene having the sequence of SEQ ID NO:
- 3 or a sequence 95% identical thereto that encodes human frataxin protein having amino acid sequence of SEQ ID NO:
- 2.
A plasmid comprising the vector genome of SEQ ID NO:
- 8 or 12, or a sequence at least 95% identical to SEQ ID NO:
- 8 or 12.
Dependencies are inferred from the claim text.