Gene therapy for treating citrullenemia
AssigneeThe Trustees of the University of Pennsylvania
InventorsJames M. Wilson, Jenny Agnes Sidrane, Lili Wang
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Abstract
Compositions and regimens useful in treating type I citrullenemia are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human Argininosuccinate Synthase 1 (ASS1).
Claims20
A recombinant adeno-associated virus (AAV) comprising an AAV capsid, and a vector genome packaged therein, the vector genome comprising an expression cassette comprising
- a promoter operably linked to
- a nucleotide sequence encoding a human argininosuccinate synthase 1 (ASS1);
- wherein the nucleotide sequence encoding the human ASS1 comprises SEQ ID NO:
- 2.
A recombinant adeno-associated virus (AAV) comprising an AAV capsid, and a vector genome packaged therein, the vector genome comprising:
- (a) an AAV 5′ ITR sequence;
- (b) a nucleotide sequence encoding a human argininosuccinate synthase 1(ASS1);
- and
- (c) an AAV 3′ ITR sequence,
- wherein the nucleotide sequence encoding the human ASS1 comprises SEQ ID NO:
- 2.
A plasmid comprising an expression cassette, the expression cassette comprising a promoter operably linked to a nucleotide sequence encoding a human argininosuccinate synthase 1 (ASS1),
- wherein the nucleotide sequence encoding the human ASS1 comprises SEQ ID NO:
- 2.
Dependencies are inferred from the claim text.