Metabolic therapy for oxidative stress in the brain through targeted neuronal catabolism of N-acetyl-aspartic acid
AssigneeRowan University
InventorsPaola Leone, Jeremy Francis
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Abstract
The present invention provides a novel method of treatment for treating brain disorders that manifest oxidative stress by providing targeted populations of neurons with the ability to catabolize the acetylated amino acid derivative, N-acetylaspatic acid (NAA) and further supply extraphysiological levels of ATP to neurons via the targeted expression of the NAA catabolic enzyme aspartoacylase (ASPA) in neurons and astrocytes.
Claims20
A method of reducing oxidative stress in a subject suffering from or at risk for Huntington's disease, the method comprising:
- (a) identifying a subject suffering from or at risk for Huntington's disease;
- and
- (b) administering to the subject an effective amount of a formulation comprising an isolated nucleic acid comprising a nucleotide sequence that encodes aspartoacylase (ASPA) under conditions whereby the nucleic acid is expressed in a host cell of the subject and whereby expression of ASPA leads to enhanced catabolism of NAA, increased concentration of AcCoA, and reduced oxidative stress.
A method for improving the disease phenotype of a subject at risk for or suffering from Huntington's disease, the method comprising administering to the subject an effective amount of a formulation comprising an isolated nucleic acid comprising a nucleotide sequence that encodes ASPA under conditions whereby the nucleic acid is expressed in a host cell of the subject and whereby expression of ASPA leads to enhanced catabolism of NAA, increased concentration of AcCoA, and reduced oxidative stress, wherein the formulation is administered to a tissue afflicted by the disease.
Dependencies are inferred from the claim text.