Photo Courtesy of Andrea Fernandez de Soto Imagine a life where every time you step outside, everything in your environment poses a life-threatening risk. For children born with ADA-SCID (adenosine deaminase severe combined immunodeficiency), this is a reality. Their immune systems are so compromised that a common cold or a simple scrape on the knee … Continue reading Gene Therapy Brings Hope to Families Affected by Rare Immune Disorder
ADA-SCID
Bubble baby treatment cleared to restart clinical trial
Evie Vaccaro: Photo courtesy Nancy Ramos Three families battling a life-threatening immune disorder got some great news last week. A clinical trial that could save the life of their child has once again been given the go-ahead by the US Food and Drug Administration (FDA). The clinical trial is the work of UCLA’s Dr. Don … Continue reading Bubble baby treatment cleared to restart clinical trial
Looking back and looking forward: good news for two CIRM-supported studies
https://open.spotify.com/episode/4IimCtQ8STpFikwZRXqSAB Dr. Rosa Bacchetta on the right with Brian Lookofsky (left) and Taylor Lookofsky after CIRM funded Dr. Bacchetta's work in October 2019. Taylor has IPEX syndrome It’s always lovely to end the week on a bright note and that’s certainly the case this week, thanks to some encouraging news about CIRM-funded research targeting blood … Continue reading Looking back and looking forward: good news for two CIRM-supported studies
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
CIRM-funded therapy helps “bubble babies” lead a normal life
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
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
Stem Cell Agency’s supporting role in advancing research for rare diseases
The recent agreement transferring GSK’s rare disease gene therapies to Orchard Therapeutics was good news for both companies and for the patients who are hoping this research could lead to new treatments, even cures, for some rare diseases. It was also good news for CIRM, which played a key role in helping Orchard grow to … Continue reading Stem Cell Agency’s supporting role in advancing research for rare diseases
The Stem Cellar 