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US 12,716,065 B2Granted
Antagonism as a therapy for TDP-43 proteinopathies
AssigneeUniversity of Southern California
InventorsJustin Ichida, Gabriel Linares
Life of this patent
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Abstract
Disclosed is a method of treating a subject who has a neurological disease. In one aspect, the method includes a step of administering an effective dose of a SYF2 antisense or inhibitory nucleic acid to a subject in need thereof, thereby restoring nuclear localization of TDP-43.
Claims24
A method of treating a human subject having a neurological disease, the method including the step of administering to the subject an effective dose of a human SYF2 antisense molecule that is complementary to the mRNA sequence encoded by the nucleic acid sequence of SEQ ID NO:
- 4, or a vector expressing the SYF2 antisense molecule, wherein (i) the SYF2 antisense molecule is 12 to 30 nucleotides in length and (ii) the neurological disease is selected from the group consisting of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
Dependencies are inferred from the claim text.