Gene Therapy Shows Promise in Treating Deadly Brain Disorders

A novel ‘gene-silencing’ drug could be the key to treating two devastating neurological disorders, spinocerebellar ataxia type 2 (SCA2) and amyotrophic lateral sclerosis (ALS).SCA2 is an inherited disorder that inflicts damage on the brain’s cerebellum, causing patients to have issues with balance, coordination, walking and similar movements. ALS induces degeneration of nerve cells in the brain and spinal cord making patients gradually lose their ability to perform basic functions like move, speak, eat, or breathe.There are different factors that can initiate the onset of these diseases, but two new studies indicate the first signs of a possible treatment approach for both SCA2 and ALS.Researchers from the University of Utah and Stanford University performed two separate preclinical trials with a therapy that targets the ataxin-2 gene, both yielding novel results in mouse models.

Source: Gene Therapy Shows Promise in Treating Deadly Brain Disorders

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