Compositions useful in treatment of cdkl5 deficiency disorder (cdd)
AssigneeThe Trustees of the University of Pennsylvania
InventorsJames M. Wilson, Ralf Schmid
Life of this patent
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Abstract
Provided is a recombinant adeno-associated virus (rAAV) having an AAV capsid and a vector genome which comprises a nucleic acid sequence encoding a functional CDKL5 (hCDKLK5). Also provided are a production system useful for producing the rAAV, a pharmaceutical composition comprising the rAAV, and a method of treating a subject having CDD, or ameliorating symptoms of CDD, or delaying progression of CDD via administrating an effective amount of the rAAV to a subject in need thereof.
Claims18
A recombinant adeno-associated virus (rAAV) useful for treating CDKL5 deficiency disorder (CDD), wherein the rAAV comprises:
- (a) an AAVhu68 or AAVrh91 capsid;
- and
- (b) a vector genome in the AAV capsid of (a), wherein the vector genome comprises a 5′ AAV inverted terminal repeat (ITR), an expression cassette comprising a human CDKL5 sequence of nucleotides 1 to 2883 of SEQ ID NO:
- 22 operably linked to regulatory sequences which direct expression thereof and which further comprise four tandem miR183 targeting sequences, and a 3′ AAV ITR.
14-15. (canceled)
A nucleic acid molecule useful for producing an rAAV vector comprising a vector genome comprising a 5′ AAV inverted terminal repeat (ITR), an expression cassette comprising a human CDKL5 sequence of nucleotides 1 to 2883 of SEQ ID NO:
- 22 operably linked to regulatory sequences which direct expression thereof and which further comprise four tandem miR183 targeting sequences, and a 3′ AAV ITR.
Dependencies are inferred from the claim text.