How could a gene that causes one type of ALS be switched off? In episode 87, Tim Miller from the Washington University in St. Louis discusses his research into therapies that target the single strands of DNA or RNA which cause many cases of amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig's disease. His article “Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS” was published with multiple co-authors on July 9th 2020 in the New England Journal of Medicine. 



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Websites and other resources







NEJM editorial on Tim's study

Tim's lab website

Video of Tim discussing new therapies for ALS:









https://www.youtube.com/watch?v=4I0asIWLCxM

 



 

Select media and press

 

EurekAlert | WBUR | Physician's Weekly | Futurity | Medscape | 2 Minute Medicine

 



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Hosts / Producers

Ryan Watkins &  Doug Leigh

How to Cite

Watkins, R., Leigh, D., & Miller, T.. (2020). Parsing Science - Silencing an ALS Gene. doi: 10.6084/m9.figshare.13232651

 

Music

What’s The Angle? by Shane Ivers

Transcript