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.

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.

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.

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.

Abstract: The present disclosure provides ICE-cleaved alpha-synuclein fragments as biomarkers for alpha-synuclein-associated disease or disorder and/or for ICE-regulator therapy.

Abstract: The present invention provides pharmaceutical compositions and related methods for stabilizing retromer in cells.

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.

Abstract: The present disclosure provides ICE-cleaved alpha-synuclein fragments as biomarkers for alpha-synuclein-associated disease or disorder and/or for ICE-regulator therapy.

Abstract: The present disclosure identifies caspase-1/ICE as a therapeutic target for ?-synuclein associated diseases and disorders. Related methods and compositions are also provided.

Abstract: The present invention provides pharmaceutical compositions and related methods for stabilizing retromer in cells.

Abstract: One aspect of the invention relates to a method of treating or preventing a neurodegenerative disease, comprising the step of administering to a patient in need thereof a therapeutically effective amount of an inhibitor of the formation of advanced glycation end products. Another aspect of the invention relates to a proteasome activity-based screening assay to select compounds which may be useful for treating or preventing a neurodegenerative disease, and the materials used therein. Yet another aspect of the invention relates to molecules, and methods of use thereof, which bind at or adjacent to SOD-I Trp32, including molecules that bind in a site adjacent to SOD-I Trp32 whether or not it is oxidized, for treating or preventing neurodegenerative disease.

Abstract: The present disclosure provides ICE-cleaved alpha-synuclein fragments as biomarkers for alpha-synuclein-associated disease or disorder and/or for ICE-regulator therapy.

Abstract: The present invention provides the surprising finding that alpha-synuclein exists in vivo as a folded tetramer. Provided are various methods and technologies that arise from this finding, including methods and kits for identifying individuals susceptible to or suffering from certain diseases, disorders or conditions associated with stability of alpha-synuclein tetramers, and/or individuals likely (or not) to respond to therapy with agents that alter level and/or stability of alpha-synuclein tetramers.

Abstract: Disclosed herein are methods and related compositions for identifying compounds that stabilize natively folded tetrameric ?-synuclein. These methods and compositions are useful for the treatment and diagnosis of ?-synuclein-associated diseases and disorders.

Abstract: One aspect of the invention relates to a method of treating or preventing a neurodegenerative disease, comprising the step of administering to a patient in need thereof a therapeutically effective amount of an inhibitor of the formation of advanced glycation end products. Another aspect of the invention relates to a proteasome activity-based screening assay to select compounds which may be useful for treating or preventing a neurodegenerative disease, and the materials used therein. Yet another aspect of the invention relates to molecules, and methods of use thereof, which bind at or adjacent to SOD-I Trp32, including molecules that bind in a site adjacent to SOD-I Trp32 whether or not it is oxidized, for treating or preventing neurodegenerative disease.

Abstract: A computational method that predicts chemical and electrostatic properties of residues in proteins and utilizes information contained in those predictions to identify various interaction sites is disclosed. The various interaction sites may include, for example, cofactor binding sites, ligand binding sites, catalytic (active) sites or recognition sites. The method of the invention identifies the ionizable residues in the protein with anomalous predicted titration behavior and searches for the clustering of those residues into putative interaction sites. Practicing the method of the invention requires only the structure of the subject protein (which may be deduced, a priori, from the amino acid sequence) and, thus, may be applied to proteins that bear no similarity in structure or sequence to any previously characterized protein.