Poster for the documentary "Ending Disease" 2020 has been, to say the very least, a difficult and challenging year for all of us. But while the focus of the world has, understandably, been on the coronavirus there was also some really promising advances in stem cell research. Those advances are captured in a great new … Continue reading Inspiring new documentary about stem cell research
Rare Diseases
“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
Positive results from CIRM-funded LAD-I trial presented at the 62nd American Society of Hematology Annual Meeting
Gaurav Shah, M.D., CEO and President of Rocket Pharmaceuticals Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease caused by a mutation in a specific gene that causes low levels of a protein called CD18. Due to low levels of CD18, the adhesion of immune cells is affected, which negatively impacts the body’s ability to … Continue reading Positive results from CIRM-funded LAD-I trial presented at the 62nd American Society of Hematology Annual Meeting
CIRM Board Approves Four New Clinical Trials
A breakdown of CIRM's clinical trials by disease area This past Thursday the governing Board of the California Institute for Regenerative Medicine (CIRM) approved four new clinical trials in addition to ten new discovery research awards. These new awards bring the total number of CIRM-funded clinical trials to 68. Additionally, these new additions have allowed … Continue reading CIRM Board Approves Four New Clinical Trials
CIRM-funded development of stem cell therapy for Canavan disease shows promising results
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
CIRM-funded treatment gets orphan drug and rare pediatric disease designations from FDA
From left to right: Brian Lookofsky , Taylor Lookofsky, and Rosa Bacchetta, M.D. Picture taken October 2019 Last year, CIRM awarded $5.53 million to Rosa Bacchetta, M.D. at Stanford University to complete the work necessary to conduct a clinical trial for IPEX syndrome. This is a rare disease caused by mutations in the FOXP3 gene, which leaves people … Continue reading CIRM-funded treatment gets orphan drug and rare pediatric disease designations from FDA
Cures, clinical trials and unmet medical needs
When you have a great story to tell there’s no shame in repeating it as often as you can. After all, not everyone gets to hear first time around. Or second or third time. So that’s why we wanted to give you another opportunity to tune into some of the great presentations and discussions at … Continue reading Cures, clinical trials and unmet medical needs
How stem cells are helping her win the fight of her life
We have all read about people who smoke a pack of cigarettes and drink a bottle of whiskey a day and somehow manage to live a long, healthy life. Then there are people like Sandra Dillon. She lived as healthy a life as you can imagine; she exercised a lot, ate a healthy diet and … Continue reading How stem cells are helping her win the fight of her life
CIRM funded trial for LAD-I announces positive results
Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease caused by a mutation in a specific gene that causes low levels of a protein called CD18. Due to low levels of CD18, the adhesion of immune cells is affected, which negatively impacts the body’s ability to combat infections. Rocket Pharmaceuticals has announced positive results from … Continue reading CIRM funded trial for LAD-I announces positive results
Partners in health
From left to right: Heather Dahlenburg, staff research associate; Jan Nolta, director of the Stem Cell Program; Jeannine Logan White, advanced cell therapy project manager; Sheng Yang, graduate student, Bridges Program, Humboldt State University, October 18, 2019. (AJ Cheline/UC Davis) At CIRM we are modest enough to know that we can't do everything by ourselves. … Continue reading Partners in health
The Stem Cellar 