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ALLELE SPECIFIC GENOME EDITING FOR HUNTINGTON'S DISEASE THERAPY

Market NeedHuntington’s Disease (HD) is an orphan neurodegenerative disease characterized by dementia, declining cognitive function, chorea that gradually spreads to all muscles, hypokinesia, rigidity, and other symptoms of psychomotor decline. Mean onset of symptoms is 30-50 years, however there is a juvenile onset form of HD that occurs under...

Published: 7/9/2019 | Inventor(s): Beverly Davidson, Alejandro Mas Monteys, Shauna Ebanks

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Category(s): Therapeutic