Methods and compositions for treating metabolic imbalance in neurodegenerative disease
AssigneeUniversity of Massachusetts
InventorsGuangping Gao, Dominic Gessler
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Abstract
In some aspects, the disclosure relates to compositions and methods useful for the diagnosis and treatment of neurodegenerative diseases, such as leukodystrophies (e.g., Canavan Disease). In some embodiments, the methods comprise administering to a subject an N-acetylaspartate (NAA)-depleting agent or an N-acetylaspartate (NAA)-depleting agent based upon the subject's metabolic profile.
Claims25
1-30. (canceled)
A method for treating a neurodegenerative disease, the method comprising:
- (a) measuring a metabolic profile of a biological sample obtained from a subject;
- (b) identifying a metabolic imbalance associated with the neurodegenerative disease based upon the metabolic profile;
- and,
- (c) administering to the subject an N-acetylaspartate (NAA)-increasing agent, wherein the metabolic imbalance comprises a decrease in N-acetylaspartate (NAA) level.
A method of increasing ATP production in a subject, the method comprising administering to a subject a recombinant adeno-associated virus (rAAV) comprising a transgene encoding ASPA enzyme or NAT8L enzyme, wherein the subject does not have an ASPA deficiency or a neurodegenerative disease.
Dependencies are inferred from the claim text.