Research Objective We propose to discovery and evaluate antisense gene therapy for specific mutations underlying debilitating or life-threatening neurodevelopmental diseases including epilepsy and autism syndromes. Impact The conditions are four…
Research Objective AAV9-Cas13 gene therapy for Angelman syndrome using a first-in-kind mechanism of action that will safely and permanently restore expression of endogenous UBE3A that is deficient in CNS neurons.…
Research Objective We will use human patient induced pluripotent stem cell (hiPSC)-based models to screen for a drug that activates a transcription factor critical to the treatment of Autism Spectrum…
Autism spectrum disorders (ASD) are complex neurodevelopmental diseases that affect about 1% of children in the United States. Such diseases are mainly characterized by deficits in verbal communication, impaired social…
Autism spectrum disorders (ASD) are a family of disabling disorders of the developing brain that affect about 1% of the population. Studying the biology of these conditions has been difficult…
People with autism have difficulty with language and social interaction as if they are trapped inside their own brains. There is no cure. Dr. Alysson Muotri of UCSD devised a…
Jen, mother of 10 year-old Milo who has a severe form of autism describes the impact of the disorder on her family and shares her thoughts about stem cell research. …
Since World War II, over 80,000 chemical compounds have been developed in the U.S. About 3,000 of these chemicals are produced at rate of one million pounds per year. Do…
Since World War II, over 80,000 chemical compounds have been developed in the U.S. About 3,000 of these chemicals are produced at rate of one million pounds per year. Do…
