Recently the CIRM Board voted to support the creation of a Rare Disease Advisory Council (RDAC) in California. An RDAC is an advisory body providing a platform for the rare community to have a stronger voice in state government. They address the needs of rare patients and families by giving stakeholders an opportunity to make … Continue reading A rare chance to help those in need
Rare Diseases
CIRM funded trial may pave way for gene therapy to treat different diseases
Image Description: Jordan Janz (left) and Dr. Stephanie Cherqui (right) According to the National Organization for Rare Disorders (NORD), a disease is consider rare if it affects fewer than 200,000 people. If you combine the over 7,000 known rare diseases, about 30 million people in the U.S. are affected by one of these conditions. A … Continue reading CIRM funded trial may pave way for gene therapy to treat different diseases
Paving the Way
When someone scores a goal in soccer all the attention is lavished on them. Fans chant their name, their teammates pile on top in celebration, their agent starts calling sponsors asking for more money. But there’s often someone else deserving of praise too, that’s the player who provided the assist to make the goal possible … Continue reading Paving the Way
New Study Shows CIRM-Supported Therapy Cures More than 95% of Children Born with a Fatal Immune Disorder
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
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
Charting a course for the future
A new home for stem cell research? Have you ever been at a party where someone says “hey, I’ve got a good idea” and then before you know it everyone in the room is adding to it with ideas and suggestions of their own and suddenly you find yourself with 27 pages of notes, all … Continue reading Charting a course for the future
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
The Stem Cellar 