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US 12,569,525 B2Granted
Gene therapy for treating phenylketonuria
AssigneeThe Trustees of the University of Pennsylvania
InventorsJames M. Wilson, Jenny Agnes Sidrane, Scott Ashley
Life of this patent
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Abstract
Compositions and regimens useful in treating phenylketonuria are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human phenylalanine hydroxylase.
Claims19
A recombinant adeno-associated virus (AAV) comprising an AAV capsid and a vector genome packaged therein, said vector genome comprising:
- (a) a nucleotide sequence comprising SEQ ID NO:
- 1 encoding a human phenylalanine hydroxylase (PAH).
A plasmid comprising an expression cassette, the expression cassette comprising a nucleotide sequence comprising SEQ ID NO:
- 1 encoding a human phenylalanine hydroxylase (PAH).
Dependencies are inferred from the claim text.