Abstract
Reducing the production of a certain protein in the brain could lead to treatments for two different neurodegenerative diseases—spinocerebellar ataxia type 2 (SCA2) and amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease—according to a pair of studies published in Nature. “I’m very excited about the papers,” says Beverly L. Davidson of Children’s Hospital of Philadelphia, who was not involved in the work. “They’re not cures but they show positive effects. And for these two disorders, that’s a good thing.” The protein that the two research teams target is ataxin-2. The exact function of ataxin-2 in the brain is not fully understood, but people with SCA2 have many extra copies of a three-nucleotide repeat, CAG, in their version of the ataxin-2 gene. Expression of the resulting mutant protein causes the loss of neurons mainly in the cerebellum, leading to difficulty in speech and in coordinating movements of the limbs
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