Gene therapy for ceroid lipofuscinoses
AssigneeThe Trustees of the University of Pennsylvania
InventorsJames M. Wilson, Nathan Katz, Christian Hinderer, Juliette Hordeaux
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Abstract
Provided herein are methods and compositions for treatment of Batten disease. Such compositions include a recombinant adeno-associated virus (rAAV), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising (a) an AAV 5′ inverted terminal repeat (ITR) sequence; (b) a promoter; (c) a CLN2 coding sequence encoding a human TPP1; (d) an AAV 3′ ITR.
Claims30
A recombinant adeno-associated virus (rAAV), comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising:
- (a) an AAV 5′ inverted terminal repeat (ITR) sequence;
- (b) a promoter;
- (c) a CLN2 coding sequence encoding a human TPP1;
- and
- (d) an AAV 3′ ITR sequence,
- wherein the CLN2 coding sequence shares 95% or greater identity with the nucleotide sequence set forth in SEQ ID NO:
- 3.
An aqueous suspension suitable for administration to subject having Batten disease, said suspension comprising an aqueous suspending liquid and a recombinant adeno-associated virus (rAAV) useful as a therapeutic for Batten disease, said rAAV having an AAV capsid, and having packaged therein a vector genome comprising:
- (a) an AAV 5′ inverted terminal repeat (ITR) sequence;
- (b) a promoter;
- (c) a CLN2 coding sequence encoding a human TPP1;
- and
- (d) an AAV 3′ ITR sequence,
- wherein the CLN2 coding sequence shares 95% or greater identity with the nucleotide sequence set forth in SEQ ID NO:
- 3.
Dependencies are inferred from the claim text.