by University of North Carolina Health Care Credit: CC0 Public Domain Scientists at the UNC School of Medicine have reported in the journal JCI Insight encouraging early tests of a gene therapy strategy against Angelman syndrome, a neurodevelopmental disorder that features poor muscle control and balance, hard-to-treat epilepsy, and intellectual disabilities. Angelman syndrome affects roughly one in every 20,000 children,...
Tag: <span>Angelman syndrome</span>
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Scientists take major step toward Angelman Syndrome gene therapy
UNIVERSITY OF NORTH CAROLINA HEALTH CARE IMAGE: LEFT: THE GENE UBE3A IS OFF IN NEURONS FROM ANGELMAN MICE. RIGHT: CRISPR USED TO TURN ON GENE, AND NEURONS CAN FIRE PROPERLY (YELLOW). CHAPEL HILL, NC – October 21, 2020 – Babies born with a faulty maternal copy of the UBE3A gene will develop Angelman syndrome, a...
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Innovative research uncovers mechanism behind epilepsy in Angelman syndrome
Innovative research uncovers mechanism behind epilepsy in Angelman syndrome SINGAPORE, 15 January 2020 – A key mechanism underlying neuronal dysfunction in Angelman syndrome (AS), a syndromic form of autism spectrum disorder, has – for the first time – been revealed through innovative research led by Duke-NUS Medical School and the National Neuroscience Institute (NNI) under...