Ja'Ceon Golden; 'cured" of SCID At CIRM we are very cautious about using the “c” word. Saying someone has been “cured” is a powerful statement but one that loses its meaning when over used or used inappropriately. However, in the case of a new study from U.C. San Francisco and St. Jude Children’s Research Hospital … Continue reading CIRM-funded therapy helps “bubble babies” lead a normal life
Blood stem cells
Stanford scientist uses CRISPR-Cas9 and stem cells to develop potential “bubble baby” therapy
Dr. Matthew Porteus, professor of pediatrics at Stanford University. Photo courtesy of Stanford Medicine. Our immune system is an important and essential part of everyday life. It is crucial for fighting off colds and, with the help of vaccinations, gives us immunity to potentially lethal diseases. Unfortunately, for some infants, this innate bodily defense mechanism … Continue reading Stanford scientist uses CRISPR-Cas9 and stem cells to develop potential “bubble baby” therapy
Promising start to CIRM-funded trial for life-threatening blood disorder
Aristotle At CIRM we are always happy to highlight success stories, particularly when they involve research we are funding. But we are also mindful of the need not to overstate a finding. To quote the Greek philosopher Aristotle (who doesn’t often make an appearance on this blog), “one swallow does not a summer make”. In … Continue reading Promising start to CIRM-funded trial for life-threatening blood disorder
Gene therapy gives patient a cure and a new lease on life
Brenden Whittaker (left), of Ohio, is a patient born with a rare genetic immune disease who was treated at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center in a CIRM funded gene therapy trial. Dr. David Williams (on right) is Brenden's treating physician.Photo courtesy of Rose Lincoln - Harvard Staff Photographer Pursuing an education can … Continue reading Gene therapy gives patient a cure and a new lease on life
Antibody effective in cure for rare blood disorders
3D illustration of an antibody binding to a designated target. Illustration created by Audra Geras. A variety of diseases can be traced to a simple root cause: problems in the bone marrow. The bone marrow contains specialized stem cells known as hematopoietic stem cells (HSCs) that give rise to different types of blood cells. As … Continue reading Antibody effective in cure for rare blood disorders
CIRM Invests in Chemotherapy-Free Approach to Rare But Deadly Childhood Disease
David Vetter, boy diagnosed with SCID Imagine being told that your seemingly healthy newborn baby has a life-threatening disease. In a moment your whole world is turned upside down. That’s the reality for families with a child diagnosed with severe combined immunodeficiency (SCID). Children with SCID lack a functioning immune system so even a simple … Continue reading CIRM Invests in Chemotherapy-Free Approach to Rare But Deadly Childhood Disease
Tips on how to be a great Patient Advocate from three of the best Advocates around
No one sets out to be a Patient Advocate. It’s something that you become because of something that happens to you. Usually it’s because you, or a loved one or a friend, becomes ill and you want to help find a treatment. Whatever the reason, it is the start of a journey that often throws … Continue reading Tips on how to be a great Patient Advocate from three of the best Advocates around
Media shine a spotlight on dodgy stem cell clinics
A doctor collects fat from a patient's back as part of an experimental stem cell procedure in Beverly Hills, Calif. on Dec. 5, 2014. (Raquel Maria Dillon / Associated Press) For several years now, we have been trying to raise awareness about the risks posed by clinics offering unproven or unapproved stem cell therapies. At … Continue reading Media shine a spotlight on dodgy stem cell clinics
Rare disease gets go-ahead to run clinical trial
Cystinosis is one of those diseases most people have never heard of and should be very grateful they haven't. It's rare - affecting only around 500 children and young adults in the US and just 2,000 people worldwide - but it's nasty. Up to now the treatments for it have been very limited. But a … Continue reading Rare disease gets go-ahead to run clinical trial
It’s not goodbye to Dr. Bert Lubin, it’s au revoir
Dr. Bert Lubin has been a fixture at UCSF Benioff Children's Hospital Oakland long before it was even called that. When he started there 43 years ago it was just a small community hospital and through his commitment to helping those in need he has helped build it into a remarkable institution. Over the years … Continue reading It’s not goodbye to Dr. Bert Lubin, it’s au revoir
The Stem Cellar 