Video: Can CRISPR/Cas9 help cure Angelman syndrome?

July 11, 2018, University of North Carolina at Chapel Hill School of Medicine

We all have a gene called UBE3A, and when the copy we inherit from our mother works normally, our brain develops properly.

When it doesn't, the result is Angelman syndrome, a neuro-genetic disorder that mainly affects the nervous system and includes symptoms such as severe intellectual and , seizures, and problems with speech, balance, movement, and sleep.

The UNC School of Medicine lab of Mark Zylka, PhD, director of the UNC Neuroscience Center and W.R. Kenan, Jr. Distinguished Professor of Cell Biology and Physiology, has figured out exactly how to edit the genome so that the copy of UBE3A we inherit from our fathers can replace the version we inherit from our mothers. 

Explore further: Researchers account for the complex symptoms of Angelman syndrome

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