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Researchers from DZNE and the Hertie Institute for Clinical Brain Research (HIH) at the University of Tübingen, together with experts from Bonn, London and Portugal, have developed and successfully tested in human neurons cultured in the lab a new approach for treating the rare neurodegenerative disease spinocerebellar ataxia type 3 (SCA3).

Their findings, published in the journal Science Translational Medicine, could pave the way for a therapy that alleviates the disease's symptoms and slows its progression.

SCA3, also known as "Machado-Joseph disease," is a severe inherited brain disorder that can cause difficulties with walking, speech, eye movements and other symptoms.

The research team's concept is based on custom-designed drugs known as antisense oligonucleotides (ASOs). These are intended to specifically reduce the levels of a disease-causing protein.

"The disorder is triggered by a harmful form of a protein called ataxin-3. This form arises from a disease-causing change in the corresponding gene: A particular stretch of DNA is repeated too many times," explains Dr. Stefan Hauser, a research group leader at DZNE and HIH.

"Humans generally have two copies of this gene, and in SCA3, usually only one carries the disease-causing change. The other copy continues to produce normal ataxin-3, which performs important functions in the cell.

"Our goal is therefore to develop a treatment that reduces the amount of harmful ataxin-3 while largely preserving the normal protein.

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