Methods to treat mucopolysaccharidosis type II or deficiency in iduronate-2-sulfatase using a recombinant adeno-associated virus (AAV) vector encoding iduronate-2-sulfatase
AssigneesRegents of the University of Minnesota, REGENXBIO Inc.
InventorsR. Scott McIvor, Lalitha R. Belur, Walter Low, Carolyn Fairbanks, Karen Kozarsky
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Abstract
A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.
Claims25
A method to treat one or more symptoms of mucopolysaccharidosis type II (MPSII) in a human having MPSII, consisting of:
- administering to a cisterna magna of the human a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding iduronate-2-sulfatase in a physiologically compatible buffer effective to treat the one or more symptoms of MPSII.
A method to treat one or more neurological symptoms of mucopolysaccharidosis type II (MPSII) in a human having MPSII, consisting of:
- administering to a cisterna magna of the human a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 vector comprising an open reading frame encoding iduronate-2-sulfatase effective to treat the one or more symptoms of MPSII.
A method to treat one or more symptoms of mucopolysaccharidosis type II (MPSII) in a human having MPSII, consisting of:
- administering to a cisterna magna of the human a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding iduronate-2-sulfatase effective to inhibit or treat the one or more symptoms of MPSII and administering an effective amount of an immune suppressant.
A method to treat one or more symptoms associated with a deficiency in iduronate-2-sulfatase in a human, consisting of:
- providing a human with a deficiency in iduronate-2-sulfatase that is immunotolerized to iduronate-2-sulfatase;
- and administering to a cisterna magna of the human a composition comprising an amount of a rAAV9 or rAAVrh10 vector comprising an open reading frame encoding iduronate-2-sulfatase effective to treat the one or more symptoms associated with the deficiency in iduronate-2-sulfatase.
Dependencies are inferred from the claim text.