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Episode 160 BEaTS Research Radio - Using molecular scissors to fix a gene mutation in Spinal Muscular Atrophy
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Spinal Muscular Atrophy (SMA) is a devastating genetic disease that causes muscle weakness and atrophy (shrinking of muscles) in children. There is currently no cure for this disease; however, base editing, a type of gene therapy, provides potential. Dr. Rasthmi Kothary is the Deputy Scientific Director and Senior Scientist at the Ottawa Hospital Research Institute. His lab focuses on understanding the mechanisms that underlie muscle and nervous system integrity. In this episode, Dr. Kothary gives us a greater insight on what SMA is, how it is currently being treated, and his research so far on a new base editing system to potentially cure this disease.
Episode produced & hosted by: Nicole Chu
Learn more: https://www.ohri.ca/profile/rkothary
Chapters
1. Episode 160 BEaTS Research Radio - Using molecular scissors to fix a gene mutation in Spinal Muscular Atrophy (00:00:00)
2. What is spinal muscular atrophy (SMA)? (00:01:15)
3. While there have been successful therapeutics developed for SMA, why are scientists exploring base editing? (00:05:10)
4. Dr. Kothary's development of a base editing system for SMA, in collaboration with team from Harvard University (00:06:20)
5. Current challenges of using base editing as a gene therapy? (00:13:40)
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