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US 12,491,235 B2Granted
Methods and compositions for treating metabolic imbalance in neurodegenerative disease
AssigneeUniversity of Massachusetts
InventorsGuangping Gao, Dominic Gessler
Life of this patent
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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.
Claims20
An isolated nucleic acid molecule, comprising a promoter operably linked to a transgene encoding aspartoacylase (ASPA), wherein the transgene comprises the nucleic acid sequence of SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.