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A new molecule identified that controls cerebellar communication

Written by Dr. Ambika Tewari Edited by Dr. Sriram Jayabal Targeting phosphatases in the cerebellum can correct miscommunication in multiple models of ataxia. The cerebellum is essential for motor coordination and consists of the coordinated activity of different types of cells. Purkinje cells are one of the most fascinating cell Read More…

Discovery of a new molecular pathway in spinocerebellar ataxia 17

Written by Dr. Sriram Jayabal Edited by Dr. Ray Truant A potential new pathway for SCA17: gene therapy that in mice restores a critical protein deficit protects brain cells from death in SCA17. Neurodegenerative ataxias are a group of brain disorders that progressively affect one’s ability to make fine coordinated Read More…

A promising biomarker to track disease progression in SCA3

Written by Dr. Ambika Tewari Edited by Dr. Gulin Oz Neurofilament light chain could provide a reliable readout of how far an SCA3 patient’s disease has progressed How often have you heard that the most effective way to treat a disorder is early intervention? In reality, “early” is not possible Read More…

Sunrise of Gene Therapy for Friedreich’s Ataxia

Written by Dr. Marija Cvetanovic   Edited by Dr. Ronald Buijsen Researchers from the University of California show they can “edit” the Frataxin gene in human cells from Friedreich’s Ataxia and transplant them into mice. This lays the groundwork for this method to be tested for safety. Friedreich’s ataxia is a Read More…

Repeat interruptions are associated with epileptic seizures in SCA10

Written by Dr Hannah Shorrock  Edited by Larissa Nitschke Repeat interruptions in SCA10 influence repeat tract stability and are associated with epileptic seizures Multiple spinocerebellar ataxias (SCAs) are caused by repeat expansion mutations, but in some cases, these repeat expansions are interrupted. The presence of repeat interruptions can influence disease Read More…

Targeting protein degradation to alleviate symptoms in MJD

Written by Ambika Tewari   Edited by Brenda Toscano Márquez Trehalose, a natural autophagy inducer shows promise as a therapeutic candidate for MJD/SCA3 Every cell has an elaborate set of surveillance mechanisms to ensure optimal functioning. As proteins are synthesized, errors can occur leading to misfolded proteins. These abnormal proteins can Read More…

A New Use for Old Drugs

Written by Dr. Amy Smith-Dijak Edited by Logan Morrison Basic biology helps identify a new treatment for ataxia Drug design doesn’t always have to start with a blank slate. Sometimes understanding how existing drugs work can help researchers to design new ones, or even to recombine old drugs in new Read More…

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