“Mini-brains” model an autism spectrum disorder and help test treatments

Alysson Muotri, PhD, professor and director of the Stem Cell Program at UC San Diego School of Medicine and member of the Sanford Consortium for Regenerative Medicine. Image credit: UC San Diego Health Rett syndrome is a rare form of autism spectrum disorder that impairs brain development and causes problems with movement, speech, and even … Continue reading “Mini-brains” model an autism spectrum disorder and help test treatments

Stem cell gene therapy combination could help children battling a rare genetic disorder

Hunter syndrome is devastating. It’s caused by a single enzyme, IDS, that is either missing or malfunctioning. Without the enzyme the body is unable to break down complex sugar molecules and as those build up they cause permanent, progressive damage to the body and brain and, in some instances, result in severe mental disabilities. There … Continue reading Stem cell gene therapy combination could help children battling a rare genetic disorder