SRA: Preclinical Investigation of SBT Discovery Compounds in Upper Motor Neuron Cell Based Model of ALS

Project: Research project

Project Details


To investigate putative neuroprotective effects of two discovery stage mitochondrial therapies in a primary cell culture model of ALS. These studies will be performed in primary cells derived from the prp-TDP-43A315T-UeGFP mouse model developed in the Ozdinler lab (Gautam, et al. Acta Neuropathol. 2019 Jan; 137(1): 47–69). Briefly, these mice express a pathogenic human TDP43 transgene which leads to severe structural alterations in the mitochondria, nucleus and endoplasmic reticulum on the background of an eGFP reporter mouse. Both transgene (prp) and reporter (UCHL1) are driven by tissue specific promoter elements, leading to the generation of fluorescent green corticospinal motor neurons carrying the TDP43 mutation. Utilizing the endogenous fluorescence of the eGFP reporter, corticospinal motor neurons can be directly visualized in situ or purified for ex vivo analyses using fluorescent activated cell sorting.
Effective start/end date8/1/197/31/23


  • Stealth BioTherapeutics Inc. (Agmt 06/23/21 AMD 3)


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