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
bubble baby disease
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
Video: Behind the scenes of a life-saving gene therapy stem cell treatment
“We were so desperate. When we heard about this treatment were willing to do anything to come here.” In the above quote from Zahraa El Kerdi, “here” refers to UCLA, a world away from her hometown in Lebanon. In September 2015, Zahree gave birth to a son, Hussein, who appeared perfectly healthy. But by six … Continue reading Video: Behind the scenes of a life-saving gene therapy stem cell treatment
Using the AIDS virus to help children battling a deadly immune disorder
More than 35 million people around the world have been killed by HIV, the virus that causes AIDS. So, it’s hard to think that the same approach the virus uses to infect cells could also be used to help children battling a deadly immune system disorder. But that’s precisely what researchers at UC San Francisco … Continue reading Using the AIDS virus to help children battling a deadly immune disorder
Progress to a Cure for Bubble Baby Disease
Welcome back to our “Throwback Thursday” series on the Stem Cellar. Over the years, we’ve accumulated an arsenal of exciting stem cell stories about advances towards stem cell-based cures for serious diseases. Today we’re featuring stories about the progress of CIRM-funded clinical trials for the treatment of a devastating, usually fatal, primary immune disease that … Continue reading Progress to a Cure for Bubble Baby Disease
Saving Ronnie: Stem Cell & Gene Therapy for Fatal Bubble Baby Disease [Video]
During this second week of the Month of CIRM, we’ve been focusing on the people who are critical to accomplishing our mission to accelerate stem cell treatments to patients with unmet medical needs. These folks include researchers, like Clive Svendsen and his team at Cedars-Sinai Medical Center who are working tirelessly to develop a stem … Continue reading Saving Ronnie: Stem Cell & Gene Therapy for Fatal Bubble Baby Disease [Video]
From trauma to treatment: a Patient Advocate’s journey from helping her son battle a deadly disease to helping others do the same
For every clinical trial CIRM funds we create a Clinical Advisory Panel or CAP. The purpose of the CAP is to make recommendations and provide guidance and advice to both CIRM and the Project Team running the trial. It’s part of our commitment to doing everything we can to help make the trial a success … Continue reading From trauma to treatment: a Patient Advocate’s journey from helping her son battle a deadly disease to helping others do the same
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
Raising awareness about Rare Disease Day
One of the goals we set ourselves at CIRM in our 2016 Strategic Plan was to fund 50 new clinical trials over the next five years, including ten rare or orphan diseases. Since then we have funded 13 new clinical trials including four targeting rare diseases (retinitis pigmentosa, severe combined immunodeficiency, ALS or Lou Gehrig’s … Continue reading Raising awareness about Rare Disease Day
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
The Stem Cellar 