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US 12,201,697 B2Granted
AAV-IDUA vector for treatment of MPS I-associated blindness
AssigneeThe University of North Carolina at Chapel Hill
InventorsMatthew Louis Hirsch, Richard Jude Samulski
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Abstract
This invention relates to viral vectors for delivery of alpha-L-iduronidase to the cornea of a subject and methods of using the same for treatment and prevention of corneal clouding and blindness in a subject due to mucopolysaccharidosis I.
Claims17
A recombinant nucleic acid comprising a sequence encoding human alpha-L-iduronidase (IDUA), wherein the nucleotide sequence has been codon-optimized for expression in human cells, and wherein the recombinant nucleic acid comprises a nucleotide sequence at least 90% identical to SEQ ID NO:1.
Dependencies are inferred from the claim text.