LISTEN TO AN AUDIO VERSION OF THIS BLOG Adrienne Shapiro and Marissa Cors are a remarkable pair by any definition. The mother and daughter duo share a common bond, and a common goal. And they are determined not to let anyone stop them achieving that goal. Marissa was born with sickle cell disease (SCD) a … Continue reading Mother and daughter team up to fight bias and discrimination in treatment for people with sickle cell disease
Gene editing
Celebrating a young life that almost wasn’t
Often on the Stem Cellar we feature CIRM-funded work that is helping advance the field, unlocking some of the secrets of stem cells and how best to use them to develop promising therapies. But every once in a while it’s good to remind ourselves that this work, while it may often seem slow, is already … Continue reading Celebrating a young life that almost wasn’t
City of Hope researchers discover potential therapy to treat brain tumors
Glioblastoma (GBM) is a common type of aggressive brain tumor that is found in adults. Survival of this type of brain cancer is poor with just 40% survival in the first-year post diagnosis and 17% in the second year, according to the American Association of Neurological Surgeons. This disease has taken the life of former … Continue reading City of Hope researchers discover potential therapy to treat brain tumors
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
A new way to evade immune rejection in transplanting cells
Immune fluorescence of HIP cardiomyocytes in a dish; Photo courtesy of UCSF Transplanting cells or an entire organ from one person to another can be lifesaving but it comes with a cost. To avoid the recipient’s body rejecting the cells or organ the patient has to be given powerful immunosuppressive medications. Those medications weaken the … Continue reading A new way to evade immune rejection in transplanting cells
An Open Letter to CIRM for World Sickle Cell Day
Nancy M. Rene Dear CIRM, World Sickle Cell Day is this Saturday June 19th. The goal of this day is to increase knowledge of the disease and understanding of the challenges faced. It is a day that I greet with very mixed feelings. I’m of course extremely grateful to CIRM for the time and money … Continue reading An Open Letter to CIRM for World Sickle Cell Day
CIRM-catalyzed spinout files for IPO to develop therapies for genetic diseases
Graphite Bio, a CIRM-catalyzed spinout from Stanford University that launched just 14 months ago has now filed the official SEC paperwork for an initial public offering (IPO). The company was formed by CIRM-funded researchers Matt Porteus, M.D., Ph.D. and Maria Grazia Roncarolo, M.D. Six years ago, Dr. Porteus and Dr. Roncarolo, in conjunction with Stanford … Continue reading CIRM-catalyzed spinout files for IPO to develop therapies for genetic diseases
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
New Study Shows CIRM-Supported Therapy Cures More than 95% of Children Born with a Fatal Immune Disorder
Dr. Donald B. Kohn; Photo courtesy UCLA A study published in the New England Journal of Medicine shows that an experimental form of stem cell and gene therapy has cured 48 of 50 children born with a deadly condition called ADA-SCID. Children with ADA-SCID, (severe combined immunodeficiency due to adenosine deaminase deficiency) lack a key … Continue reading New Study Shows CIRM-Supported Therapy Cures More than 95% of Children Born with a Fatal Immune Disorder
CIRM funding helps improve immune cell therapy to combat HIV
Image description: T cell infected with HIV. Image Credit: National Institute of Allergy and Infectious Diseases (NIAID) In June of last year we wrote about how Dr. Scott Kitchen and his team at UCLA are engineering blood forming stem cells in order to fight HIV, a potentially deadly virus that attacks the immune system and … Continue reading CIRM funding helps improve immune cell therapy to combat HIV