Tackling ALS through gene therapy
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that has no cure. Recently scientists have successfully used the CRISPR-Cas9 gene editing tool to remove the mutated SOD1 gene in a mouse model that mimics ALS. This video uses a combination of interviews and drawings to educate the general public about ALS and CRISPR. Dr. John Turnbull is a professor of Neurology at McMaster University and a neurologist and director of the ALS Clinic at Hamilton Health Sciences. Dr. John Turnbull conducts ALS research and speaks about ALS in this video. Vladimir Ljubicic is an assistant professor of kinesiology and Canada Research Chair (Tier 2) in Neuromuscular Plasticity in Health and Disease. Ljubicic's laboratory studies the mechanisms that regulate the maintenance and remodelling of the neuromuscular system in health and disease.
This video was made by McMaster University Demystifying Medicine students Farah Al Faraj, Sarah Qureishi, Ingrid Kao and Neel Patel.
Copyright McMaster University 2018
References:
ALS Society of Canada (2018). What is ALS? Retrieved from
https://www.als.ca/about-als/what-is-als/
Bunton-Stasyshyn, R. K. A., Saccon, R. A., Fratta, P., & Fisher, E. M. C. (2015). SOD1 Function and Its Implications for Amyotrophic Lateral
Sclerosis Pathology: New and Renascent Themes. The Neuroscientist: A Review Journal Bringing Neurobiology, Neurology and Psychiatry, 21(5), 519–529.
Gaj, T., Ojala, D. S., Ekman, F. K., Byrne, L. C., Limsirichai, P., & Schaffer, D. V. (2017). Science Advances, 3(12), eaar3952
Ralph, G. S., Radcliffe, P. A., Day, D. M., Carthy, J. M., Leroux, M. A., Lee, D. C. P., … Azzouz, M. (2005). Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nature Medicine, 11(4), 429–433. https://doi.org/10.1038/nm1205
Rosen, D. R., Siddique, T., Patterson, D., Figlewicz, D. A., Sapp, P., Hentati, A., … Brown, R. H. (1993). Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis. Nature, 362(6415), 59–62. https://doi.org/10.1038/362059a0
