TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS
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Abstract
Nonsense-mediated mRNA decay (NMD) polypeptides, nucleic acids encoding NMD polypeptides, and methods of using such polypeptides and nucleic acids in the treatment of ALS and in screening for agents for the treatment of ALS are described.
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19 Claims
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1. (canceled)
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2. A method of treating amyotrophic lateral sclerosis (ALS) in a human subject in need thereof, the method comprising:
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administering to the human subject a therapeutically effective amount of a UPF1 polypeptide or a nucleic acid encoding a UPF1 polypeptide; wherein the human subject does not have a mutation in a SOD1 gene and wherein the UPF1 polypeptide comprises the amino acid sequence of SEQ ID NO;
2. - View Dependent Claims (3, 4, 7, 8, 17, 18, 19)
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5. The method of claim 5, wherein the vector is a viral vector.
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6. The method of claim 6, wherein the viral vector is a retroviral vector, a lentiviral vector, an adenoviral vector, or an adeno-associated viral vector.
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9. A method of reducing FUS/TLS-mediated neuronal cytotoxicity in a human subject in need thereof, the method comprising:
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administering to the human subject a therapeutically effective amount of a UPF1 polypeptide or a nucleic acid encoding a UPF1 polypeptide, thereby reducing the FUS/TLS-mediated neuronal cytotoxicity in the human subject; wherein the UPF1 polypeptide comprises the amino acid sequence of SEQ ID NO;
2. - View Dependent Claims (10, 11, 12, 13, 14, 15, 16)
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Specification