Expression vector for cholesterol 24-hydrolase in therapy of amyotrophic lateral sclerosis
AssigneeInserm (Institut National De La Sante Et De La Recherche Medicale)
InventorsFrançoise PIGUET, Nathalie CARTIER-LACAVE
Life of this patent
Estimating the term…
Abstract
The present invention relates to a vector for use in the treatment of amyotrophic lateral sclerosis associated, or not associated, with fronto temporal dementia and related motoneuron disorders, which vector comprises cholesterol 24-hydroxylase encoding nucleic acid.
Claims21
A method of treating or preventing amyotrophic lateral sclerosis (ALS) in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of a vector comprising a nucleic acid that encodes cholesterol 24-hydroxylase in expressible form.
(canceled)
A method for treating or preventing a motoneuron disorder in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of a vector comprising a nucleic acid that encodes cholesterol 24-hydroxylase in expressible form to thereby reduce p62 aggregates in motoneurons of the subject.
A method for reducing p62 aggregates in motoneurons of a subject in need thereof, comprising administering to the subject an effective amount of a vector comprising a nucleic acid that encodes cholesterol 24-hydroxylase in expressible form, to thereby express the cholesterol 24-hydroxylase in motoneurons of the subject.
Dependencies are inferred from the claim text.