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US 2026/0077060 A1Published application
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 an adenosine triphosphate (ATP) level in the nervous system of a subject in need thereof, comprising:
- administering to the subject a therapeutically effective amount of a composition that comprises a nucleic acid encoding aspartoacylase (ASPA) or a functional fragment thereof,
- wherein the ASPA or functional fragment thereof (A) comprises an amino acid sequence at least 75% identical to the sequence of SEQ ID NO:
- 1 and (B) has the enzymatic activity of aspartoacylase.
A method for treating 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 brain or the spinal cord or the brain stem of the subject a therapeutically effective amount of a composition comprising a nucleic acid encoding ASPA or a functional fragment thereof,
- wherein the ASPA or functional fragment thereof (A) comprises an amino acid sequence at least 75% identical to the sequence of SEQ ID NO:
- 1 and (B) has the enzymatic activity of aspartoacylase.
2 more elements
Dependencies are inferred from the claim text.