AANS Neurosurgeon | Volume 28, Number 3, 2019


University of Michigan Researchers Use Gene Silencing to Alleviate Common Ataxia

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In what researchers are calling a game changer for future ataxia treatments, a new study showed the ability to turn down the disease progression of the most common dominantly inherited ataxia.

Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease, affects about one in 20,000 people. There’s no disease-modifying therapy available, and patients slowly progress to an early death. A single gene mutation causes this neurodegenerative disease, making it an ideal target for a group of University of Michigan researchers.

For this preclinical study, University of Michigan researchers employed nucleotide-based gene silencing to target the SCA3 disease gene, ATXN3. They greatly reduced levels of the mutant RNA coded by the gene in a mouse model of the disease without any toxic effects.

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