Brandeis University

Dagmar Ringe

Shulin Ju

Greg Petsko

Gregory A Petsko

Jeffrey N Agar

Quyen Q Hoang

Walter R P Novak

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.

Methods of reducing FUS/TLS- or TDP-43-mediated neuronal cytotoxicity in amyotrophic lateral sclerosis (ALS) by UPF2

Methods of reducing FUS/TLS- or TDP-43-mediated neuronal cytotoxicity by UPF1

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

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

Ice-cleaved alpha-synuclein as a biomarker

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.

Compositions and methods for the diagnosis, treatment, and prevention of amyotrophic lateral sclerosis and related neurological diseases

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Cambridge, MA, United States of America

Dagmar Ringe