GENE THERAPY FOR TREATMENT OF MUCOPOLYSACCHARIDOSIS IIIA
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United States of America
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Issued Date -
Feb 6, 2025
app pub date -
Nov 11, 2022
filing date -
Nov 11, 2022
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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 hSGSH, optionally comprising stabilizing amino acid changes, a regulatory sequence which direct expression of hSGSH 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 IIIA.
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