One of the great pleasures of my job is getting to meet the high school students who take part in our SPARK or Summer Internship to Accelerate Regenerative Medicine Knowledge program. It's a summer internship for high school students where they get to spend a couple of months working in a world class stem cell … Continue reading The present and future of regenerative medicine
Genes + Cells
Tiny tools for the smallest of tasks, editing genes
YOU CAN LISTEN TO THIS BLOG AS AN AUDIOCAST ON SPOTIFY Developing new tools to edit genes Having the right tools to do a job is important. Try using a large screwdriver to tighten the screws on your glasses and you quickly appreciate that it’s not just the type of tool that’s important, it’s also … Continue reading Tiny tools for the smallest of tasks, editing genes
Gene therapy is life-changing for children with a life-threatening brain disorder
If you have never heard of AADC deficiency count yourself lucky. It’s a rare, incurable condition that affects only around 135 children worldwide but it’s impact on those children and their families is devastating. The children can’t speak, can’t feed themselves or hold up their head, they have severe mood swings and often suffer from … Continue reading Gene therapy is life-changing for children with a life-threatening brain disorder
CIRM funded trial may pave way for gene therapy to treat different diseases
Image Description: Jordan Janz (left) and Dr. Stephanie Cherqui (right) According to the National Organization for Rare Disorders (NORD), a disease is consider rare if it affects fewer than 200,000 people. If you combine the over 7,000 known rare diseases, about 30 million people in the U.S. are affected by one of these conditions. A … Continue reading CIRM funded trial may pave way for gene therapy to treat different diseases
Stem cell gene therapy for Fabry disease shows positive results in patients
Darren Bidulka rests after his modified blood stem cells were transplanted into him at the Foothills Medical Centre in Calgary in 2017, allowing him to stop his enzyme therapy. (From left): Dr. Jeffrey Medin, Medical College of Wisconsin, Dr. Aneal Khan, the experimental trial lead in Calgary, and Darren Bidulka. Image Credit: Darren Bidulka Fabry … Continue reading Stem cell gene therapy for Fabry disease shows positive results in patients
First patient in CIRM funded X-CGD trial gives back by working in patient care
Brenden Whittaker Brenden Whittaker was born with a rare genetic disorder called X-linked chronic granulomatous disease (X-CGD). This condition affects the immune system's ability to fight off common germs, specifically bacteria and fungi, and can result in infections that would only be mild for healthy people. Unfortunately for Brenden, he has suffered life-threatening infections that … Continue reading First patient in CIRM funded X-CGD trial gives back by working in patient care
CIRM supported study of gene silencer blocks ALS degeneration, saves motor function
Dr. Martin Marsala, UC San Diego Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease, is a neurodegenerative disease that destroys the nerve cells in the brain and spinal cord. As a result of ALS, the motor neurons that enable bodily movement and muscle control are harmed, which can make it difficult to move, … Continue reading CIRM supported study of gene silencer blocks ALS degeneration, saves motor function
One family’s fight to save their son’s life, and how stem cells made it possible
CIRM's mission is very simple: to accelerate stem cell treatments to patients with unmet medical needs. Anne Klein's son, Everett, was a poster boy for that statement. Born with a fatal immune disorder Everett faced a bleak future. But Anne and husband Brian were not about to give up. The following story is one Anne … Continue reading One family’s fight to save their son’s life, and how stem cells made it possible
CIRM-funded therapy helps “bubble babies” lead a normal life
Ja'Ceon Golden; 'cured" of SCID At CIRM we are very cautious about using the “c” word. Saying someone has been “cured” is a powerful statement but one that loses its meaning when over used or used inappropriately. However, in the case of a new study from U.C. San Francisco and St. Jude Children’s Research Hospital … Continue reading CIRM-funded therapy helps “bubble babies” lead a normal life
Gene therapy gives patient a cure and a new lease on life
Brenden Whittaker (left), of Ohio, is a patient born with a rare genetic immune disease who was treated at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center in a CIRM funded gene therapy trial. Dr. David Williams (on right) is Brenden's treating physician.Photo courtesy of Rose Lincoln - Harvard Staff Photographer Pursuing an education can … Continue reading Gene therapy gives patient a cure and a new lease on life
The Stem Cellar 