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
Rare Diseases
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
How stem cells are helping her win the fight of her life
We have all read about people who smoke a pack of cigarettes and drink a bottle of whiskey a day and somehow manage to live a long, healthy life. Then there are people like Sandra Dillon. She lived as healthy a life as you can imagine; she exercised a lot, ate a healthy diet and … Continue reading How stem cells are helping her win the fight of her life
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
Battling COVID and turning back the clock on stem cell funding
Coronavirus Battling the virus that causes COVID-19 is something that is top of everyone’s mind right now. That’s why CIRM is funding 17 different projects targeting the virus. But one of the most valuable tools in helping develop vaccines against a wide variety of diseases in the past is now coming under threat. We’ll talk … Continue reading Battling COVID and turning back the clock on stem cell funding
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
Exploring tough questions, looking for answers
COVID-19 and social and racial injustice are two of the biggest challenges facing the US right now. This Thursday, October 8th, we are holding a conversation that explores finding answers to both. The CIRM Alpha Stem Cell Clinic Network Symposium is going to feature presentations about advances in stem cell and regenerative research, highlighting treatments … Continue reading Exploring tough questions, looking for answers
CIRM Bridges program prepared student for research of a rare disease
Ian Blong, Ph.D., CIRM San Francisco State University Bridges to Stem Cell Research Alumnus Recently, The New York Times released a powerful article that tells the stories of four different families navigating the challenges of having a family member with a rare disease. One of these stories focused on Matt Wilsey, a tech entrepreneur and … Continue reading CIRM Bridges program prepared student for research of a rare disease
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