Treatment of amyotrophic lateral sclerosis
AssigneeBrandeis University
InventorsGreg Petsko, Dagmar Ringe, Shulin Ju
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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.
Claims18
1-52. (canceled)
A method of reducing TAR-DNA-binding protein 43 (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, the method comprising:
- administering to the subject a composition comprising a UPF2 polypeptide or a nucleic acid encoding the UPF2 polypeptide, thereby reducing the TDP-43 mediated neuronal cytotoxicity in the subject,
- wherein the UPF2 polypeptide comprises the amino acid sequence of SEQ ID NO:4.
The method of claim 07, wherein the viral vector is a retroviral vector, a lentiviral vector, an adenoviral vector, or an adeno-associated viral vector.
A method of reducing fused in sarcoma/translocated in sarcoma (FUS/TLS)- or TAR-DNA-binding protein 43 (TDP-43)-mediated neuronal cytotoxicity, the method comprising:
- providing to a neuronal or glial cell a composition comprising a UPF2 polypeptide or a nucleic acid encoding a UPF2 polypeptide, thereby reducing the FUS/TLS- or TDP-43-mediated neuronal cytotoxicity in the neuronal or glial cell;
- wherein the UPF2 polypeptide comprises the amino acid sequence of SEQ ID NO:4.
Dependencies are inferred from the claim text.