FGSL Webinar: Genetic modifier screen in human neurons reveals interaction between SARM1 and rare neuropathies

Join the Functional Genomics Screening Laboratory (FGSL) team as they welcome Dr Andrea Elser for this special webinar.

Speaker:
Dr. Andrea Elser
Applied Stem Cell Sciences Team, Centre for Genomics Research, Discovery Sciences, BioPharmaceuticals R&D, AstraZeneca, Gothenburg, Sweden.

Abstract: Rare neuropathies, chemotherapy-induced neurodegeneration and SARM1 activity share common characteristics. NAD-consuming enzyme SARM1 plays a central role in programmed axon death and its loss-of-function has been shown to be protective in subtypes of Charcot-Marie-Tooth disease and Hereditary Spastic Paraplegia. These diseases are characterised by neurite degeneration and mitochondrial defects and currently lack effective therapies. SARM1 inhibitors may provide a much-needed disease-modifying opportunity for certain mechanistic subtypes of rare neurological disorders. To evaluate if SARM1 loss-of-function can protect against neurite degeneration linked to rare neuropathies, we performed arrayed phenotypic CRISPR-Knock-out (KO) screens in wildtype and SARM1-KO human iPSC-derived neurons, focusing on 60 disease-associated genes. In this webinar, we will present in vitro model validation data and how the genetic screen was used to identify rare neuropathies where SARM1 inhibition can be neuroprotective.