Gene therapy for mucopolysaccharidosis type IIIA

摘要

A recombinant AAV (rAAV) containing an AAV capsid and a vector genome packaged therein, wherein the vector genome is an AAV5'terminal inversion sequence (ITR), an engineered nucleic acid sequence encoding a functional hSGSH, a target. Recombinant AAV (rAAV), including a control sequence that directs the expression of hSGSH in cells, and AAV3'ITR, are provided herein. Also provided is a pharmaceutical composition comprising rAAV described herein in a formulation buffer, and a method of treating a human subject diagnosed with MPS IIIA. [Selection diagram] No diagram