When we launched our Facebook Live “Ask the Expert” series earlier this year we wanted to create an opportunity for people to hear from and question experts about specific diseases or disorders. The experts we turned to were medical ones, neurologists and neuroscientists in the case of the first two Facebook Live events, stroke and … Continue reading What makes an expert an expert?
Sickle cell disease
CIRM funded study results in the first ever in utero stem cell transplant to treat alpha thalassemia
Imagine being able to cure a genetic disorder before a baby is even born. Thanks to a CIRM funded study, what would have been a mere dream a couple of years ago has become a reality. Drs. Tippi MacKenzie and Juan Gonzalez Velez of the University of California San Francisco (UCSF) have successfully treated alpha … Continue reading CIRM funded study results in the first ever in utero stem cell transplant to treat alpha thalassemia
Stem Cell Agency Heads to Inland Empire for Free Patient Advocate Event
I am embarrassed to admit that I have never been to the Inland Empire in California, the area that extends from San Bernardino to Riverside counties. That’s about to change. On Monday, April 16th CIRM is taking a road trip to UC Riverside, and we’re inviting you to join us. We are holding a special, … Continue reading Stem Cell Agency Heads to Inland Empire for Free Patient Advocate Event
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
Stem Cell Agency invests in stem cell therapies targeting sickle cell disease and solid cancers
Today CIRM’s governing Board invested almost $10 million in stem cell research for sickle cell disease and patients with solid cancer tumors. Clinical trial for sickle cell disease City of Hope was awarded $5.74 million to launch a Phase 1 clinical trial testing a stem cell-based therapy for adult patients with severe sickle cell disease … Continue reading Stem Cell Agency invests in stem cell therapies targeting sickle cell disease and solid cancers
Just a Mom: The Journey of a Sickle Cell Disease Patient Advocate [video]
Adrienne Shapiro will tell you that she’s just a mom. And it’s true. She is just a mom. Just a mom who is the fourth generation of mothers in her family to have children born with sickle cell disease. Just a mom who was an early advocate of innovative stem cell and gene therapy research … Continue reading Just a Mom: The Journey of a Sickle Cell Disease Patient Advocate
A new study suggests CRISPR gene editing therapies should be customized for each patient
You know a scientific advance is a big deal when it becomes the main premise and title of a Jennifer Lopez-produced TV drama. That’s the case for CRISPR, a revolutionary gene-editing technology that promises to yield treatments for a wide range of genetic diseases. In fact, clinical trials using the CRISPR method are already underway … Continue reading A new study suggests CRISPR gene editing therapies should be customized for each patient
CIRM-Funded Clinical Trials Targeting Blood and Immune Disorders
This blog is part of our Month of CIRM series, which features our Agency’s progress towards achieving our mission to accelerate stem cell treatments to patients with unmet medical needs. This week, we’re highlighting CIRM-funded clinical trials to address the growing interest in our rapidly expanding clinical portfolio. Today we are featuring trials in our blood and … Continue reading CIRM-Funded Clinical Trials Targeting Blood and Immune Disorders
World Sickle Cell Day: A View from the Front Line
June 19th is World Sickle Cell Day. Sickle cell disease is an inherited blood disorder that causes normally round red blood cells to take on an abnormal sickle shape, resulting in clogged arteries, severe pain, increased risk of stroke and reduced life expectancy. To mark the occasion we asked Nancy M. Rene to write a … Continue reading World Sickle Cell Day: A View from the Front Line
Translating great stem cell ideas into effective therapies
In science, there are a lot of terms that could easily mystify people without a research background; “translational” is not one of them. Translational research simply means to take findings from basic research and advance them into something that is ready to be tested in people in a clinical trial. Yesterday our Governing Board approved … Continue reading Translating great stem cell ideas into effective therapies