Yanhong Shi, Ph.D., City of Hope Canavan disease is a fatal neurological disorder, the most prevalent form of which begins in infancy. It is caused by mutation of the ASPA gene, resulting in the deterioration of white matter (myelin) in the brain and preventing the proper transmission of nerve signals. The mutated ASPA gene causes … Continue reading CIRM-funded development of stem cell therapy for Canavan disease shows promising results
Gene editing
CIRM-funded treatment gets orphan drug and rare pediatric disease designations from FDA
From left to right: Brian Lookofsky , Taylor Lookofsky, and Rosa Bacchetta, M.D. Picture taken October 2019 Last year, CIRM awarded $5.53 million to Rosa Bacchetta, M.D. at Stanford University to complete the work necessary to conduct a clinical trial for IPEX syndrome. This is a rare disease caused by mutations in the FOXP3 gene, which leaves people … Continue reading CIRM-funded treatment gets orphan drug and rare pediatric disease designations from FDA
A look back at 15 years of CIRM funding at UCLA
Researchers in the lab of CIRM grantee Donald Kohn, M.D. Image Credit: UCLA Broad Stem Cell Center Since the first grant was issued in April 2006, CIRM has funded a wide range of research conducted by top scientists at UCLA for a wide range of diseases. To give a retrospective look at all the research, … Continue reading A look back at 15 years of CIRM funding at UCLA
Cures, clinical trials and unmet medical needs
When you have a great story to tell there’s no shame in repeating it as often as you can. After all, not everyone gets to hear first time around. Or second or third time. So that’s why we wanted to give you another opportunity to tune into some of the great presentations and discussions at … Continue reading Cures, clinical trials and unmet medical needs
CIRM funded trial for LAD-I announces positive results
Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease caused by a mutation in a specific gene that causes low levels of a protein called CD18. Due to low levels of CD18, the adhesion of immune cells is affected, which negatively impacts the body’s ability to combat infections. Rocket Pharmaceuticals has announced positive results from … Continue reading CIRM funded trial for LAD-I announces positive results
Partners in health
From left to right: Heather Dahlenburg, staff research associate; Jan Nolta, director of the Stem Cell Program; Jeannine Logan White, advanced cell therapy project manager; Sheng Yang, graduate student, Bridges Program, Humboldt State University, October 18, 2019. (AJ Cheline/UC Davis) At CIRM we are modest enough to know that we can't do everything by ourselves. … Continue reading Partners in health
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
Remembering a stem cell pioneer in the fight against HIV/AIDS
Timothy Ray Brown. Photo courtesy Seattle Times Timothy Ray Brown, a man who was the first person to be cured of HIV, giving hope to millions of people around the world, died at his home in Palm Springs this week. He was just 54 years old. For years Brown was known simply as “the Berlin … Continue reading Remembering a stem cell pioneer in the fight against HIV/AIDS
It’s all about the patients
Ronnie, born with a fatal immune disorder now leading a normal life thanks to a CIRM-funded stem cell/gene therapy: Photo courtesy of his mum Upasana Whenever you are designing something new you always have to keep in mind who the end user is. You can make something that works perfectly fine for you, but if … Continue reading It’s all about the patients
Charting a new course for stem cell research
What are the latest advances in stem cell research targeting cancer? Can stem cells help people battling COVID-19 or even help develop a vaccine to stop the virus? What are researchers and the scientific community doing to help address the unmet medical needs of underserved communities? Those are just a few of the topics being … Continue reading Charting a new course for stem cell research
The Stem Cellar 