Methods for the treatment of neurodegenerative disorders
AssigneeTakeda Pharmaceuticals U.S.A., Inc.
InventorsNessan Anthony BERMINGHAM, Alan BUCKLER, Irina Ariane ANTONIJEVIC, Brian R. BETTENCOURT
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Abstract
The present disclosure features useful compositions and methods for treatment of neurodegenerative disorders, e.g., in a subject in need thereof.
Claims192
A method of treating, preventing, or delaying the onset and/or progression of a neurodegenerative disorder in a subject in need thereof, the method comprising administering a therapeutically effective amount of a pharmaceutically acceptable composition comprising one or more oligonucleotide molecules, that is 15-30 linked nucleotides in length, or a pharmaceutically acceptable salt thereof, to the subject by intraceberebroventricular (“ICV”) injection.
The method of claim 1 or claim 2, wherein the neurodegenerative disease is not a triplet repeat disorder.
A method of reducing the amount of a target mRNA in a cell, the method comprising contacting the cell with one or more oligonucleotide molecule that is 15-30 linked nucleotides in length, or a pharmaceutically acceptable salt thereof, for a time sufficient to obtain degradation of the target mRNA, wherein the target mRNA is derived from a target gene selected from the group consisting of MAPT, APP, LRRK2, SNCA, HTT, ATXN1, ATXN2, ATXN3, PSEN1, PSEN2, ATN1, HSPA2, C4A, C4B, C9orf72, and/or TDP43.
Dependencies are inferred from the claim text.