Dr. Donald B. Kohn; Photo courtesy UCLA A study published in the New England Journal of Medicine shows that an experimental form of stem cell and gene therapy has cured 48 of 50 children born with a deadly condition called ADA-SCID. Children with ADA-SCID, (severe combined immunodeficiency due to adenosine deaminase deficiency) lack a key … Continue reading New Study Shows CIRM-Supported Therapy Cures More than 95% of Children Born with a Fatal Immune Disorder
Rare Diseases
Positive results for patients enrolled in CIRM-funded trial of a rare pediatric disease
Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease that prevents patients from combating infections. This leads to recurring bacterial and fungal infections that respond poorly to antibiotics, require frequent hospitalizations, and can be fatal. It is caused by a mutation in a specific gene that causes low levels of a protein called CD18. The … Continue reading Positive results for patients enrolled in CIRM-funded trial of a rare pediatric disease
A little history in the making by helping the tiniest patients
Dr. Diana Farmer stands with Dr. Aijun Wang and their UC Davis research team. It’s appropriate that at the start of Women’s History Month, UC Davis’ Dr. Diana Farmer is making a little history of her own. She launched the world’s first clinical trial using stem cells to treat spina bifida before the child is … Continue reading A little history in the making by helping the tiniest patients
Stem cell gene therapy for Fabry disease shows positive results in patients
Darren Bidulka rests after his modified blood stem cells were transplanted into him at the Foothills Medical Centre in Calgary in 2017, allowing him to stop his enzyme therapy. (From left): Dr. Jeffrey Medin, Medical College of Wisconsin, Dr. Aneal Khan, the experimental trial lead in Calgary, and Darren Bidulka. Image Credit: Darren Bidulka Fabry … Continue reading Stem cell gene therapy for Fabry disease shows positive results in patients
A word from our Chair, several in fact
In 2005, the New Oxford American Dictionary named “podcast" its word of the year. At the time a podcast was something many had heard of but not that many actually tuned in to. My how times have changed. Now there are some two million podcasts to chose from, at least according to the New York … Continue reading A word from our Chair, several in fact
A Month of CIRM: Where we’ve been, where we’re going
All this month we are using our blog and social media to highlight a new chapter in CIRM’s life, thanks to the voters approving Proposition 14. We are looking back at what we have done since we were created in 2004, and also looking forward to the future. We kick off this event with a … Continue reading A Month of CIRM: Where we’ve been, where we’re going
Inspiring new documentary about stem cell research
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
“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