Methods of treating or preventing amyotrophic lateral sclerosis
AssigneeRowan University
InventorsJeremy Francis, Paola Leone
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Abstract
This disclosure provides methods for treating, ameliorating, or reversing at least one symptom of amyotrophic lateral sclerosis (ALS) in a subject by increasing the amount of neuronal aspartate in spinal cord through administration of a therapeutically effective amount of a composition comprising a nucleic acid encoding ASPA or a functional fragment thereof.
Claims20
A method of increasing the amount of neuronal aspartate in the brain and spinal cord or increasing the N-acetylaspartate (NAA) catabolism in the brain or spinal cord to facilitate mitochondrial function in a subject at risk of developing amyotrophic lateral sclerosis (ALS) or suffering from ALS comprising identifying said subject by screening the subject for ALS and administering to the subject a therapeutically effective amount of a composition, wherein the composition comprises a nucleic acid encoding aspartoacylase (ASPA) or a functional fragment thereof, wherein the ASPA or functional fragment thereof comprises an amino acid sequence that is at least 75% identical to the sequence of SEQ ID NO:1 carried on a recombinant adeno-associated virus (rAAV) vector, and wherein the ASPA or functional fragment thereof has the enzymatic activity of aspartoacylase.
A method of treating, ameliorating, or reversing at least one symptom of amyotrophic lateral sclerosis (ALS) in a subject having ALS, comprising the steps of:
- a) identifying a patient in need of an increase in intracellular aspartate level;
- and
- b) administering to the subject a therapeutically effective amount of a composition that increases a level or activity of aspartoacylase (ASPA) in motor neurons of the subject.
A kit for increasing a level or activity of ASPA in a cell of a subject comprising a rAAV1 vector or a virus-like particle, wherein the virus or the virus-like particle comprises a nucleic acid encoding ASPA or a functional fragment thereof, wherein the ASPA or functional fragment thereof comprises an amino acid sequence that is at least 75% identical to the sequence of SEQ ID NO:
- 1, and wherein the ASPA or functional fragment thereof has the enzymatic activity of aspartoacylase.
Dependencies are inferred from the claim text.