Gene therapy for mucopolysaccharidosis IIIB
AssigneeThe Trustees of the University of Pennsylvania
InventorsJames M. Wilson, Christian Hinderer, Juliette Hordeaux
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Abstract
Provided herein is a recombinant AAV (rAAV) comprising an AAV capsid and a vector genome packaged therein, wherein the vector genome comprises an AAV 5′ inverted terminal repeat (ITR), an engineered nucleic acid sequence encoding a functional human N-acetyl-alpha-glucosaminidase (hNAGLU), a regulatory sequence which direct expression of hNAGLU in a target cell, and an AAV 3′ ITR. Also provided is a pharmaceutical composition comprising a rAAV as described herein in a formulation buffer, and a method of treating a human subject diagnosed with MPS IIIB.
Claims28
A recombinant AAV (rAAV) comprising an AAV capsid and a vector genome packaged therein, wherein the vector genome comprises an AAV 5′ inverted terminal repeat (ITR), an engineered nucleic acid sequence encoding a functional human N-acetyl-alpha-glucosaminidase (hNAGLU), a regulatory sequence which directs expression of hNAGLU in a target cell, and an AAV 3′ ITR, wherein the hNAGLU coding sequence is at least 99% identical to SEQ ID NO:
- 1.
A vector comprising an engineered nucleic acid sequence encoding a functional hNAGLU and a regulatory sequence which directs expression thereof in a target cell, wherein the hNAGLU coding sequence is at least 99% identical to SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.