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
SCID
Rare Disease Day – fighting for awareness and hope
It’s hard thinking of something as rare when one in 20 people are at risk of experiencing it in their lifetime. But that’s the situation with rare diseases. There are more than 7,000 of them and each affects under 200,000 people. In some cases they may only affect a few hundred people. But for each … Continue reading Rare Disease Day – fighting for awareness and hope
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
71 for Proposition 71
Proposition 71 is the state ballot initiative that created California's Stem Cell Agency. This month, the Agency reached another milestone when the 71st clinical trial was initiated in the CIRM Alpha Stem Cell Clinics (ASCC) Network. The ASCC Network deploys specialized teams of doctors, nurses and laboratory technicians to conduct stem cell clinical trials at … Continue reading 71 for Proposition 71
Stories that caught our eye: SanBio’s Traumatic Brain Injury trial hits its target; A new approach to endometriosis; and a SCID kid celebrates Halloween in style
Hopeful signs for treating brain injuries There are more than 200,000 cases of traumatic brain injury (TBI) in the US every year. The injuries can be devastating, resulting in everything from difficult sleeping to memory loss, depression and severe disability. There is no cure. But this week the SanBio Group had some encouraging news from … Continue reading Stories that caught our eye: SanBio’s Traumatic Brain Injury trial hits its target; A new approach to endometriosis; and a SCID kid celebrates Halloween in style
California’s Stem Cell Agency Accelerates Treatments to Patients
The following article is an Op Ed that appeared in today's print version of the San Francisco Chronicle Biotechnology was born in California in the 1970s based on the discovery out of one of its universities and California is responsible for an industry that has impacted the lives of billions of people worldwide. In 2004, … Continue reading California’s Stem Cell Agency Accelerates Treatments to Patients
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 Story of a South African Bubble Boy and a Gene Therapy That Gave Him His Life Back
Ayaan Isaacs was born in South Africa on March 4th, 2016 as a seemingly healthy baby. But only a few days in to life, he contracted a life-threatening liver infection. He thankfully survived, only to have the doctors discover a few weeks later that he had something much more troubling – a rare disease that … Continue reading The Story of a South African Bubble Boy and a Gene Therapy That Gave Him His Life Back
Breaking the isolation of rare diseases
How can something that affects 30 million Americans, one in ten people in the US, be called rare? But that’s the case with people who have a rare disease. There are around 7,000 different diseases that are categorized as rare because they affect fewer than 200,000 people. Less than five percent of these diseases have … Continue reading Breaking the isolation of rare diseases
The Stem Cellar 