Can CRISPR/Cas9 help cure Angelman Syndrome?

In this UNC Science Short video, we feature the work of Mark Zylka's lab, which the Angelman Syndrome Foundation has awarded $200,000 to investigate CRISPR/Cas9 gene editing technology as the basis for a cure to Angelman Syndrome, a rare but devastating neuro-genetic condition.

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 developmental disabilities, 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. 

Watch our latest UNC Science Short to learn how the Zylka Lab is using CRISPR/Cas9 gene editing technology as the basis for a one-time treatment to cure people with Angelman Syndrome. This work is funded through a grant from the Angelman Syndrome Foundation.

Zylka is also a member of the UNC Autism Research Center.

Watch more UNC Science Shorts from the UNC Health Care/UNC School of Medicine communications team.

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