AAV-SGSH Vectors for Treatment of Mucopolysaccharidosis IIIA
AssigneeThe University of North Carolina at Chapel Hill
InventorsHaiyan Fu, Tierra Bobo
Life of this patent
Estimating the term…
Abstract
This invention relates to viral vectors for delivery of N-sulfoglucosamine sulfohydrolase (SGSH) to a subject. In some aspects, the SGSH sequence is optimized for expression in human cells. The invention further relates to methods of using the vector to increase secretion of SGSH from a cell and for treatment and prevention of mucopolysaccharidosis IIIA.
Claims24
A recombinant nucleic acid comprising a sequence encoding human N-sulfoglucosamine sulfohydrolase (SGSH) that is codon-optimized for expression in human cells, wherein the recombinant nucleic acid comprises a nucleotide sequence at least 90% identical to SEQ ID NO:1.
(canceled)
22-25. (canceled)
Dependencies are inferred from the claim text.