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 (rAAV) useful as a liver-directed therapeutic for citrullinemia, said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising:
- (a) an AAV 5′ inverted terminal repeat (ITR);
- (b) a promoter;
- (c) a coding sequence encoding a human argininosuccinate synthase 1 (ASS1);
- (d) an AAV 3′ ITR,
- wherein the coding sequence of (c) is SEQ ID NO:
- 2.
An aqueous suspension suitable for administration to a citrullinemia patient, said suspension comprising an aqueous suspending liquid and about 1×1012 genome copies (GC)/mL to about 1×1014 GC/mL of a rAAV useful as a liver-directed therapeutic for citrullinemia, said rAAV having an AAV capsid, and having packaged therein a vector genome comprising:
- (a) an AAV 5′ ITR;
- (b) a promoter;
- (c) a coding sequence encoding an ASS1;
- and
- (d) an AAV 3′ ITR,
- wherein the coding sequence of (c) is SEQ ID NO:
- 2.
Dependencies are inferred from the claim text.