ADENO-ASSOCIATED VIRUS MEDIATED GENE TRANSFER TO THE CENTRAL NERVOUS SYSTEM
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.
1 - 21 . (canceled)
22 . A method to inhibit or treat one or more symptoms of ceroid lipofuscinosis in a mammal in need thereof, comprising:
administering to a cisterna magna of the mammal in need thereof a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding a lysosomal enzyme effective to inhibit or treat the one or more symptoms of ceroid lipofuscinosis.
23 . A method to inhibit or treat one or more symptoms of mucopolysaccharoidosis (MPS) type III in a mammal in need thereof, comprising:
administering to a cisterna magna of the mammal in need thereof a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding heparan sulfate sulfatase effective to inhibit or treat the one or more symptoms of MPS type III.
24 . A method to inhibit or treat one or more symptoms of MPS type III in a mammal in need thereof, comprising:
administering to a cisterna magna of the mammal in need thereof a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding N-acetyl-alpha-D-glucosaminidase effective to inhibit or treat the one or more symptoms of MPS type III.
25 . A method to inhibit or treat one or more symptoms of MPS type VI in a mammal in need thereof, comprising:
administering to a cisterna magna of the mammal in need thereof a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding arylsulfatase B effective to inhibit or treat the one or more symptoms of MPS type VI.
26 . A method to inhibit or treat one or more symptoms of Morquio syndrome (MPS type IV) in a mammal in need thereof, comprising:
administering to a cisterna magna of the mammal in need thereof a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding Beta galactosidase or galactosamine-6-sulfate sulfatase effective to inhibit or treat the one or more symptoms of MPS type IV.
27 . A method to inhibit or treat one or more symptoms of Gaucher disease in a mammal in need thereof, comprising:
administering to a cisterna magna of the mammal in need thereof a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding glucocerebrosidase. effective to inhibit or treat the one or more symptoms of Gaucher disease.
28 . A method to inhibit or treat one or more symptoms associated with a deficiency of glucocerebrosidase or dopamine decarboxylase in a mammal, comprising:
administering to a cisterna magna of the mammal in need thereof a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding glucocerebrosidase.or dopamine decarboxylase effective to inhibit or treat the one or more symptoms associated with the deficiency.
29 . A method to inhibit or treat one or more symptoms of Pompe Disease in a mammal in need thereof, comprising:
administering to a cisterna magna of the mammal in need thereof a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding acid maltase effective to inhibit or treat the one or more symptoms of Pompe Disease.
30 . A method to inhibit or treat one or more symptoms of Metachromatic leukodystrophy in a mammal in need thereof, comprising:
administering to a cisterna magna of the mammal in need thereof a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding Aryl sulfatase A effective to inhibit or treat the one or more symptoms of Metachromatic leukodystrophy.
31 . A method to inhibit or treat one or more symptoms of MPS type VII in a mammal in need thereof, comprising:
administering to a cisterna magna of the mammal in need thereof a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding beta-glucuronidase effective to inhibit or treat the one or more symptoms of MPS type VII.
32 . A method to inhibit or treat one or more symptoms of MPS type VIII in a mammal in need thereof, comprising:
administering to a cisterna magna of the mammal in need thereof a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding glucosamine-6-sulfate sulfatase effective to inhibit or treat the one or more symptoms of MPS type VIII.
33 . A method to inhibit or treat one or more symptoms of MPS type IX in a mammal in need thereof, comprising:
administering to a cisterna magna of the mammal in need thereof a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding hyaluronidase effective to inhibit or treat the one or more symptoms of MPS type IX.
34 . A method to inhibit or treat one or more symptoms of Niemann-Pick disease in a mammal in need thereof, comprising:
administering to a cisterna magna of the mammal in need thereof a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding sphingomyelinase effective to inhibit or treat the one or more symptoms of Niemann-Pick disease.