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
Disease Areas
Stem Cell Roundup: better model of schizophrenia, fasting boosts stem cells, and why does our hair gray.
Stem cell photo of the week: Recreating brain cell interactions for studying schizophrenia Our pick for the stem cell image of the week is from the laboratory of Rusty Gage at the Salk Institute. The team generated multiple types of nerve cells from stem cells to more closely represent the interactions that occur in the … Continue reading Stem Cell Roundup: better model of schizophrenia, fasting boosts stem cells, and why does our hair gray.
New CIRM Alpha Stem Cell Clinic offers HOPE for boys with deadly disease
For people battling Duchenne Muscular Dystrophy (DMD), a rare and fatal genetic disorder that slowly destroys muscles, hope has often been in short supply. There is no cure and treatments are limited. But now a new clinical trial at the site of one of the newest CIRM Alpha Stem Cell Clinic Network members could change … Continue reading New CIRM Alpha Stem Cell Clinic offers HOPE for boys with deadly disease
Livers skip stem cells, build missing structures from scratch via direct cell identity conversion
Stem cells…eh, who needs them anyway?! That’s what you might be thinking after today, at least for some forms of liver disease. That’s because a team of researchers from UCSF and Cincinnati Children’s Hospital Medical Center just published results in Nature showing liver cells can directly change identity, or transdifferentiate, in order to build, from … Continue reading Livers skip stem cells, build missing structures from scratch via direct cell identity conversion
Meet the high school student who moonlights as a neuroscientist
As part of our CIRM scholar blog series, we’re featuring the research and career accomplishments of CIRM funded students. Today, you’ll read about one of our former SPARK high school students. Emma Friedenberg is a high school senior at Campbell Hall in North Hollywood, California. She’s also an up-and-coming neuroscientist who has her sights set … Continue reading Meet the high school student who moonlights as a neuroscientist
Therapies Targeting Cancer, Deadly Immune Disorder and Life-Threatening Blood Condition Get Almost $32 Million Boost from CIRM Board
An innovative therapy that uses a patient’s own immune system to attack cancer stem cells is one of three new clinical trials approved for funding by CIRM's Governing Board. Researchers at the Stanford University School of Medicine were awarded $11.9 million to test their Chimeric Antigen Receptor (CAR) T Cell Therapy in patients with B … Continue reading Therapies Targeting Cancer, Deadly Immune Disorder and Life-Threatening Blood Condition Get Almost $32 Million Boost from CIRM Board
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
Patients at the heart of Alpha Stem Cell Clinics Symposium
I have been to a lot of stem cell conferences over the years and there’s one recent trend I really like: the growing importance and frequency of the role played by patient advocates. There was a time, not so long ago, when having a patient advocate speak at a scientific conference was almost considered a … Continue reading Patients at the heart of Alpha Stem Cell Clinics Symposium
Stem Cell Roundup: The brain & obesity; iPSCs & sex chromosomes; modeling mental illness
Stem Cell Image of the Week: Obesity-in-a-dish reveals mutations and abnormal function in nerve cells Our stem cell image of the week looks like the work of a pre-historic cave dweller who got their hands on some DayGlo paint. But, in fact, it’s a fluorescence microscopy image of stem cell-derived brain cells from the lab … Continue reading Stem Cell Roundup: The brain & obesity; iPSCs & sex chromosomes; modeling mental illness
Straight to brain: A better approach to ALS cell therapies?
Getting the go ahead to begin a clinical trial by no means marks an end to a research team’s laboratory studies. A clinical trial is merely one experiment and is designed to answer a specific set of questions about a specific course of treatment. There will inevitably be more questions to pursue back in the … Continue reading Straight to brain: A better approach to ALS cell therapies?