Audio Interview

Translational research in amyotrophic lateral sclerosis (ALS)

Published on May 30, 2018   25 min

Other Talks in the Category: Pharmaceutical Sciences

Interviewer: Doctor Gitler, thank you for sparing the time today. We're going to be talking about Amyotrophic Lateral Sclerosis, ALS. I'm going to be asking your opinions on therapeutic interventions, the conditions necessary to decide whether and when to attempt the various approaches that can be considered, and the likely timescale for commencing development of candidate interventions. So, just to start, what do you think of the opportunities presented by recent research? Prof. Gitler: There have been many exciting new developments in ALS research, and I think the most exciting new ones focus on human genetics. So it's the discovery of mutations in genes that cause ALS. And some of those discoveries have directly led to therapeutic strategies. Two specific examples, the first one is the discovery in 1993 of mutations in the SOD1 gene as a cause of familial ALS, and much research over the years has provided evidence that what those mutations do is to cause some sort of toxicity to the protein that the SOD1 gene encodes. And that realization has suggested that therapies that lower

Translational research in amyotrophic lateral sclerosis (ALS)

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