Yanhong Shi, Ph.D., City of Hope Canavan disease is a fatal neurological disorder, the most prevalent form of which begins in infancy. It is caused by mutation of the ASPA gene, resulting in the deterioration of white matter (myelin) in the brain and preventing the proper transmission of nerve signals. The mutated ASPA gene causes … Continue reading CIRM-funded development of stem cell therapy for Canavan disease shows promising results
Canavan disease
Stem Cell Stories That Caught our Eye: Duchenne muscular dystrophy and short telomeres, motor neurons from skin, and students today, stem cell scientists tomorrow
Short telomeres associated with Duchenne Muscular Dystrophy. Duchenne Muscular Dystrophy (DMD) is a severe muscle wasting disease that typically affects young men. There is no cure for DMD and the average life expectancy is 26. These are troubling facts that scientists at the University of Pennsylvania are hoping to change with their recent findings in … Continue reading Stem Cell Stories That Caught our Eye: Duchenne muscular dystrophy and short telomeres, motor neurons from skin, and students today, stem cell scientists tomorrow
Rare disease underdogs come out on top at CIRM Board meeting
It seems like an oxymoron but one in ten Americans has a rare disease. With more than 7,000 known rare diseases it’s easy to see how each one could affect thousands of individuals and still be considered a rare or orphan condition. Only 5% of rare diseases have FDA approved therapies People with rare … Continue reading Rare disease underdogs come out on top at CIRM Board meeting
The Stem Cellar 