Rapid Flow Cytometry Screen for Identifying Novel ALS Drug Leads
Abstract
Amyotrophic lateral sclerosis (ALS) arises due to proteins misfolding inside motor neuron cells, leading to toxicity, cell death and loss of motor function. TDP-43 is an important protein known to misfold, leading to its clumping or "aggregating" in the cytoplasm to form large insoluble deposits (inclusions), which are associated with cell death and thereby causally associated with ALS pathology. This project uses a motor neuron cell model in which fluorescently-tagged TDP-43 forms cytoplasmic inclusions, in a high-throughput drug screen of thousands of chemicals to find potential drugs to treat ALS patients. A small number of "hits" have already been identified and will be screened in animal models of ALS, to identify a therapeutic to treat ALS patients.
Document Details
- Document Type
- Technical Report
- Publication Date
- Dec 01, 2023
- Accession Number
- AD1225474
Entities
People
- Adam Walker
- Angela Laird
- Mark R. Wilson
Organizations
- University of Wollongong