NIH researchers engineer potential drug for ALS

A gene-silencing drug displayed efficacy in the treatment of two fatal neurological diseases, according to two studies published in the journal Nature.

For one study, researchers discovered mutations in the ataxin 2 gene caused spinocerebellar ataxia type 2 — a genetic disorder that damages the cerebellum and causes difficulties with coordination and movement. The team then sought to treat the fatal condition with a drug designed to silence the gene. Injecting the drug into the brains of mice afflicted with the condition significantly reduced issues linked to the disorder.

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In a companion study, researchers used the same therapy to treat mice with amyotrophic lateral sclerosis — a neurological disease that results in paralysis and death. The treatment prevented early death in the mice with ALS.

"Surprisingly, the ataxin 2 gene may act as a master key to unlocking treatments for ALS and other neurological disorders," said Aaron Gitler, PhD, an associate professor in genetics at Stanford (Calif.) University and senior author of the second study.

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