October 31, 2018
Neuroscience
Rare Diseases
Translational Research
Grantee
A new form of therapy may halt or even reverse a form of progressive vision loss that, until now, has inevitably led to blindness. This hyper-targeted approach offers hope to individuals living with spinocerebellar ataxia type 7 (SCA7) and validates a new form of therapy with the potential to treat neurogenetic diseases effectively and with far fewer side effects than other medications.