Arsb vectors for treatment of mps vi-associated blindness and other ocular manifestations
AssigneesThe University of North Carolina at Chapel Hill, North Carolina State University
InventorsMatthew Louis Hirsch, Liujiang Song, Brian Christopher Gilger
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Abstract
This invention relates to vectors for delivery of ary lsulfatase B to the eye (e.g., cornea) of a subject and methods of using the same for treatment and pre-vention of corneal clouding and blindness in a subject due to mucopolysaccharidosis VI (MPS-VI) and other MPS VI-associated manifestations in the eye.
Claims28
A recombinant nucleic acid comprising a sequence encoding human arylsulfatase B (ARSB), wherein the nucleotide sequence has been codon-optimized for expression in human cells.
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A method of delivering ARSB to the eye of a subject, comprising administering to the eye of the subject an effective amount of a vector or polynucleotide that expresses ARSB or an ARSB polypeptide, thereby delivering ARSB to the eye of the subject.
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A method of treating, slowing the progression of, or delaying the onset of a mucopolysaccharidosis VI (MPS VI)-associated ocular manifestation in a subject in need thereof, comprising administering to the eye of the subject a therapeutically effective amount of a vector or polynucleotide that expresses ARSB or an ARSB polypeptide, thereby treating, slowing the progression of, or delaying the onset of the MPS VI-associated ocular manifestation in the subject.
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Dependencies are inferred from the claim text.