Patients with ALS, other neurological disorders look to gene-silencing drug for hope


Two recent mouse studies, led by investigators at the University of Utah and Stanford University, describe how a new antisense oligonucleotide drug was successful in reducing symptoms for both spinocerebellar ataxia type 2 (SCA2) and amyotrophic lateral sclerosis (ALS).

In the SCA2 study, Dr. Stefan Pulst, M.D., professor and chair of neurology at the University of Utah, and his team discovered that they could reduce problems associated with SCA2 by injecting mouse brains with a drug programmed to silence the ataxin 2 gene. In the accompanying study, investigators described that injections of the same type of drug into the brains of mice prevented early death and neurological problems associated with ALS, a paralyzing and often fatal disorder.

“Surprisingly, the ataxin 2 gene may act as a master key to unlocking treatments for ALS and other neurological disorders,” noted senior investigator on the ALS study Aaron Gitler, Ph.D., associate professor at Stanford University.

Dr. Gitler’s team used different mice to test the idea of combating ALS by silencing ataxin 2…The mice rapidly develop problems with walking and die early—recapitulating many of the associated symptoms of ALS patients.

[Read the original studies here and here (behind paywall)]

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post: Gene Silencing Drug Holds Potential to Treat Fatal Neurological Conditions

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