A research team at the Cedars-Sinai Regenerative Medicine Institute has been awarded a $1.9 million grant by the California institute for Regenerative Medicine. It will fund development of an induced pluripotent stem cell-based platform that will help researchers identify potential new drug candidates for spinal muscular atrophy (SMA).
SMA is caused by a genetic mutation in the SMN1 gene, which stops motor neurons manufacturing the critical protein SMN. Another gene, SMN2, does manufacture the SMN protein, but not enough is produced overall to compensate for the SMN1 gene-related deficit, the Sinai researchers explain.
Their team, led by Druv Sareen, Ph.D., aims to generate pluripotent stem cells from the skin cells of SMA patients and then trigger these to develop into motor neurons with the mutated SMN1 gene and normal SMN2 gene. This platform will then be used to test compounds for their ability to increase production of SMN by the functioning SMN2 gene.
“Pharmaceutical and biotech companies have comprehensive libraries of chemically diverse compounds, some of which could potentially be developed into drug treatments to help SMA patients,” Dr. Sareen remarks. “We will be able to test these drug libraries relatively quickly using our high-content screening approach. We hope to identify novel compounds that target the SMN2 gene specifically in patient motor neurons."