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
Genetic Disorders
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
How a tiny patch of skin helped researchers save the life of a young boy battling a deadly disease
By any standards epidermolysis bullosa (EB) is a nasty disease. It’s a genetic condition that causes the skin to blister, break and tear off. At best, it’s painful and disfiguring. At worst, it can be fatal. Now researchers in Italy have come up with an approach that could offer hope for people battling the … Continue reading How a tiny patch of skin helped researchers save the life of a young boy battling a deadly disease
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
Stem cell stories that caught our eye: bubble baby therapy a go in UK, in-utero stem cell trial and novel heart disease target
There were lots of CIRM mentions in the news this week. Here are two brief recaps written by Karen Ring to get you up to speed. A third story by Todd Dubnicoff summarizes an promising finding related to heart disease by researchers in Singapore. CIRM-funded “bubble baby” disease therapy gets special designation by UK. … Continue reading Stem cell stories that caught our eye: bubble baby therapy a go in UK, in-utero stem cell trial and novel heart disease target
Stem Cell Profiles in Courage: Brenden Whittaker
It’s not often you meet someone who says one of their favorite things in the world is mowing the lawn. But then, there aren’t many people in the world like Brenden Whittaker. In fact, as of this writing, he may be unique. Brenden was born with severe chronic granulomatous disease (x-CGD), a rare genetic disorder … Continue reading Stem Cell Profiles in Courage: Brenden Whittaker
Cured by Stem Cells
To get anywhere you need a good map, and you need to check it constantly to make sure you are still on the right path and haven’t strayed off course. A year ago the CIRM Board gave us a map, a Strategic Plan, that laid out our course for the next five years. Our Annual … Continue reading Cured by Stem Cells
Stem cell heroes: patients who had life-saving, life-changing treatments inspire CIRM Board
It’s not an easy thing to bring an entire Board of Directors to tears, but four extraordinary people and their families managed to do just that at the last CIRM Board meeting of 2016. The four are patients who have undergone life-saving or life-changing stem cell therapies that were funded by our agency. The … Continue reading Stem cell heroes: patients who had life-saving, life-changing treatments inspire CIRM Board
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
Funding stem cell research targeting a rare and life-threatening disease in children
If you have never heard of cystinosis you should consider yourself fortunate. It’s a rare condition caused by an inherited genetic mutation. It hits early and it hits hard. Children with cystinosis are usually diagnosed before age 2 and are in end-stage kidney failure by the time they are 9. If that’s not bad enough … Continue reading Funding stem cell research targeting a rare and life-threatening disease in children