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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AANS Neurosurgeon is the official socioeconomic publication of the American Association of Neurological Surgeons (AANS) and features information and analysis for contemporary neurosurgical practice. Published monthly online, AANS Neurosurgeon focuses on issues related to neurosurgery legislation, the workforce and practice management.
