Gaurav Shah, M.D., CEO and President of Rocket Pharmaceuticals 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 … Continue reading Positive results from CIRM-funded LAD-I trial presented at the 62nd American Society of Hematology Annual Meeting
CIRM
CIRM funded trial for sickle cell disease gives patient a chance for a better future
Evie Junior is participating in a CIRM funded clinical trial for sickle cell disease that uses a stem cell gene therapy approach. Image credit: UCLA Broad Stem Cell Research Center For Evie Junior, personal health and fitness have always been a top priority. During his childhood, he was active and played football, basketball, and baseball … Continue reading CIRM funded trial for sickle cell disease gives patient a chance for a better future
A guide to healing
Dr. Evan Snyder Having grown up in an era where to find your way around you had to use paper maps, a compass and a knowledge of the stars (OK, I’m not actually that old!) I’m forever grateful to whoever invented the GPS. It’s a lifesaver, and I daresay has also saved more than a … Continue reading A guide to healing
Much to be Thankful for
It's traditional this time of year to send messages of gratitude to friends and family and colleagues. And we certainly have much to be thankful for. Thanks to the voters of California, who passed Proposition 14, we have a bright, and busy, future. We have $5.5 billion to continue our mission of accelerating stem cell … Continue reading Much to be Thankful for
CIRM-funded study shows how cigarette smoke can worsen COVID-19 infection in the airways
Microscopic images of human stem cell–derived airway tissue models with cell nuclei (blue) and SARS-CoV-2 virus infected cells (green); tissue exposed to cigarette smoke (right) had 2 to 3 times more infected cells than non-exposed tissue (left).Image Credit: UCLA Broad Stem Cell Research Center/Cell Stem Cell In the middle of a pandemic, stress can run … Continue reading CIRM-funded study shows how cigarette smoke can worsen COVID-19 infection in the airways
CIRM-funded therapy to ease the impact of chemotherapy
Treatments for cancer have advanced a lot in recent years, but many still rely on the use of chemotherapy to either shrink tumors before surgery or help remove cancerous cells the surgery missed. The chemo can be very effective, but it’s also very toxic. Angiocrine Bioscience Inc. is developing a way to reduce those toxic … Continue reading CIRM-funded therapy to ease the impact of chemotherapy
Stem cell therapy for deadly childhood immune disorder goes four for four
The gold standard for any new therapy in the U.S. is approval by the Food and Drug Administration (FDA). This approval clears the therapy for sale and often also means it will be covered by insurance. But along the way there are other designations that can mean a lot to a company developing a new … Continue reading Stem cell therapy for deadly childhood immune disorder goes four for four
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
‘Mini lung’ model shows scientists early stages of new coronavirus infection
Representative image of three-dimensional human lung alveolar organoid showing alveolar stem cell marker, HTII-280 (red) and SARS-CoV-2 entry protein, ACE2 (green) Image Credit: Jeonghwan Youk, Taewoo Kim, and Seon Pyo Hong The development of organoid modeling has significantly expanded our understanding of human organs and the diseases that can affect them. For those unfamiliar with … Continue reading ‘Mini lung’ model shows scientists early stages of new coronavirus infection
The Stem Cellar 