Methods and compositions for treating metabolic imbalance
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 diseases associated with a metabolic imbalance in a subject (e.g.,cancer). In some embodiments, the methods comprise administering to a subject an N-acetylaspartate (NAA)-depleting agent or an N-acetylaspartate (NAA)-increasing agent based upon the subject's metabolic profile.
Claims49
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.
A method for treating a disease associated with a metabolic imbalance in a subject in need thereof, the method comprising administering to the subject an N-acetylaspartate (NAA)-depleting agent, wherein it has been determined that the disease is associated with a metabolic imbalance comprising a shift from beta-oxidation to glycolysis in the subject.
A method for treating a disease associated with a metabolic imbalance in a subject in need thereof, the method comprising administering to the subject an N-acetylaspartate (NAA)-depleting agent, wherein it has been determined that the disease is associated with a metabolic imbalance comprising a shift from glycolysis to beta-oxidation in the subject.
A method for treating a disease associated with a metabolic imbalance in a subject in need thereof, the method comprising administering to the subject an N-acetylaspartate (NAA)-increasing agent, wherein it has been determined that the disease is associated with a metabolic imbalance comprising a shift from beta-oxidation to glycolysis in the subject.
A method for treating a disease associated with a metabolic imbalance in a subject in need thereof, the method comprising administering to the subject an N-acetylaspartate (NAA)-increasing agent, wherein it has been determined that the disease is associated with a metabolic imbalance comprising a shift from glycolysis to beta-oxidation in the subject.
A method for treating cancer in a subject in need thereof, the method comprising:
- a. measuring a metabolic profile of a biological sample obtained from a subject;
- b. identifying a metabolic imbalance associated with cancer based upon the metabolic profile;
- and,
- c. administering to the subject an N-acetylaspartate (NAA)-depleting agent, wherein the metabolic imbalance comprises a shift from beta-oxidation to glycolysis.
The method of any one of claims 24 to 37, wherein the NAA-depleting agent is selected from the group consisting of a small molecule, a protein, and a nucleic acid.
The method of any one of claims 24 to 38, wherein the NAA-depleting agent is administered using a recombinant adeno-associated virus (rAAV).
The method of claim 38, wherein the rAAV comprises:
- (a) a capsid protein;
- and,
- (b) a nucleic acid comprising a promoter operably linked to a transgene, wherein the transgene encodes aspartoacylase (ASPA).
Dependencies are inferred from the claim text.