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US 12,673,117 B2Granted
AAV-IDS vectors for treatment of mucopolysaccharidosis II
AssigneeThe University of North Carolina at Chapel Hill
InventorsHaiyan Fu, Tierra Bobo
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Abstract
This invention relates to viral vectors for delivery of iduronate-2-sulfatase (IDS) to a subject. In some aspects the IDS sequence is optimized for expression in human cells. The invention further relates to methods of using the vector to increase secretion of IDS from a cell and for treatment and prevention of mucopolysaccharidosis II.
Claims20
A recombinant nucleic acid comprising a sequence encoding human iduronate-2-sulfatase (IDS) that is codon-optimized for expression in human cells, wherein the recombinant nucleic acid comprises a nucleotide sequence at least 99% identical to SEQ ID NO:1.
Dependencies are inferred from the claim text.