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Methods of reducing TDP-43-mediated neuronal cytotoxicity in amyotrophic lateral sclerosis by a UPF1 polypeptide or polynucleotide

  • US 10,533,038 B2
  • Filed: 10/08/2013
  • Issued: 01/14/2020
  • Est. Priority Date: 10/11/2012
  • Status: Active Grant
First Claim
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1. A method of reducing TDP-43-mediated neuronal cytotoxicity in a human subject suffering from amyotrophic lateral sclerosis (ALS), wherein the subject does not have a mutation in a SOD1 gene, comprising:

  • administering to the subject suffering from ALS a therapeutically effective amount of a UPF1 polypeptide or a nucleic acid encoding the UPF1 polypeptide, thereby reducing the TDP-43 mediated neuronal cytotoxicity in the subject;

    wherein the UPF1 polypeptide comprises the amino acid sequence of SEQ ID NO;

    2.

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