Grant: Emerging Team Grant: Regenerative Medicine and Nanomedicine - LOI

Amyotrophic lateral sclerosis (ALS) is a progressive adult-onset neurodegenerative disorder that affects primarily motor neurons in the brain and spinal cord. The disease typically culminates in paralysis and death within 3-5 years. Three thousands Canadians live with ALS. So far, most investigations on the problem of ALS have been focussed on identifying the genetic mutations of familial cases. The remaining 90% ALS patients are sporadic cases, resulting from multiple genetic and environmental factors, most of which are still unknown. There is no effective treatment for ALS and the development of therapeutics has been impaired by the lack of knowledge about the causes of sporadic ALS. A team of renowned ALS researchers are proposing to generate lines of induced pluripotent stem (iPS) cells from the skin of ALS patients and controls. From the iPS cells, they will derive motor neurons that will be investigated to identify biological defects associated with sporadic ALS. The findings will serve to discover therapeutic targets, to design iPS cell-derived bioassays for drug screening and to derive animal models for preclinical testing.