Angelman Syndrome is a severe neurodevelopmental disorder that is caused by loss of the maternal copy of the Ube3a gene. Using the gene editing technology CRISPR/Cas9, a team of scientists in the Zylka lab was able to correct the underlying molecular deficiency in the Angelman mouse model. The Zylka Lab was awarded a grant from the Angelman Syndrome Foundation to expand this finding towards a gene therapy.

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Song Lab Cover Article – Cell Stem Cell 2017

To learn more about the research, click here to watch a video created by the Angelman Syndrome Foundation.

To learn more about the Angelman Syndrome Foundation, click here.

To see other research the Zylka Lab is undertaking, click here.