Amyotrophic lateral sclerosis (ALS) is one of the most common degenerative disease of the motor neuron system. The TDP-43 pathology seems to be a dominant type of pathology across sporadic ALS types. Our TDP-43 Stress Granules Assay allows the quantification of pathological TDP43 globs into the nucleus and cytosol. This model also permits to monitor the TDP-43 protein distribution in living cells, studying the protein localization pattern in the space and time, and quantifying the fluorescence aggregation inside the cells.
For this assay, we use a stable cell line expressing fluorescent TDP-43. During the assay, cells are incubated withtest compounds and Sodium Arsenite to induce the TDP-43 aggregation. Finally, we quantify TDP43-tGFP nuclear globs using the BD Pathway HCS Reader and Attovision Compartimentalization Software.
Cell line used: TDP43-tGFP U2OS
Readout: TDP-43 Stress Granules quantification
Agonist: Sodium Arsenite
EC50 Agonist: 7.3 x 10-5 M
Positive Compound 1: Arimoclomol
Positive Compound 2: Riluzole
Assay: ALS, Amyotrophic Lateral Sclerosis, CNS, Modelos de enfermedad, Área terapéutica