METHODS FOR SLOWING FAMILIAL ALS DISEASE PROGRESSION
First Claim
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1. A method of treating amyotrophic lateral sclerosis (ALS) comprising administering to the cerebrospinal fluid of a subject in need thereof a therapeutically or prophylactically effective amount of a composition comprising:
- a modified oligonucleotide consisting of 12 to 30 linked nucleosides targeted to SOD-1, wherein the modified oligonucleotide is specifically hybridizable to SEQ ID NO;
1, anda pharmaceutically acceptable diluent or carrier.
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Abstract
Methods for slowing disease progression in an individual suffering from familial ALS are provided. Also provided are methods of increasing the survival time of an individual suffering from familial ALS. These methods employ antisense oligonucleotides targeted to SOD1, for use in inhibiting the expression of SOD1 in the central nervous system of an individual suffering from familial ALS.
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Citations
20 Claims
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1. A method of treating amyotrophic lateral sclerosis (ALS) comprising administering to the cerebrospinal fluid of a subject in need thereof a therapeutically or prophylactically effective amount of a composition comprising:
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a modified oligonucleotide consisting of 12 to 30 linked nucleosides targeted to SOD-1, wherein the modified oligonucleotide is specifically hybridizable to SEQ ID NO;
1, anda pharmaceutically acceptable diluent or carrier. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10)
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11. A method of treating amyotrophic lateral sclerosis (ALS) comprising administering to the cerebrospinal fluid of a subject in need thereof a therapeutically or prophylactically effective amount of a composition comprising:
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a modified oligonucleotide consisting of 12 to 30 linked nucleosides targeted to SOD-1, wherein the modified oligonucleotide is 100% complementary to SEQ ID NO;
1, anda pharmaceutically acceptable diluent or carrier. - View Dependent Claims (12, 13, 14, 15, 16, 17, 18, 19, 20)
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Specification