CRISPR Medicine News, 9 November 2020 | Single CRISPR-Cas9 Injection Could Cure Angelman Syndrome - CRISPR Medicine

A Single Cas9 Injection Could Mean a Lifetime of Treatment for Angelman Syndrome

A CRISPR-Cas9 treatment reactivated a crucial gene in mice and human neurons - hinting at a possible one-time therapy for Angelman syndrome. Neurobiologist Mark Zylka, who led the study, explains the science of what could be a game-changer for thousands affected by the genetic disorder that causes delayed development, problems with speech and balance and intellectual disability.
»This is pretty spectacular finding or development,« comments Dr Arthur Beaudet, a paediatrician and molecular geneticist, who is recognized as a leader in the field of genetic neurodevelopmental disorders.

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