Compositions and methods for treating amyotrophic lateral sclerosis
AssigneeResearch Institute At Nationwide Children's Hospital
InventorBrian Kaspar
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Abstract
The invention relates to pharmaceutical compositions, kits, methods, and uses for the treatment of amyotrophic lateral sclerosis. In particular, the invention relates to pharmaceutical compositions, kits, methods, and uses for the treatment of amyotrophic lateral sclerosis by decreasing the expression of a cytoplasmic granule toxin in astrocytes of a patient, or by increasing the expression of MHC class I in motor neurons of the patient.
Claims6
A method for treating a patient with amyotrophic lateral sclerosis by decreasing the expression of a cytoplasmic granule toxin in astrocytes of the patient, the method comprising the step of
- administering to the patient to treat amyotrophic lateral sclerosis a composition comprising an effective amount of a compound that decreases the expression of the cytoplasmic granule toxin in the astrocytes of the patient wherein the compound is a nucleic acid and wherein the compound inhibits the loss of motor neurons, thereby treating amyotrophic lateral sclerosis.
Dependencies are inferred from the claim text.