Dr. Matthew Porteus The US Food and Drug Administration (FDA) has granted Investigational New Drug (IND) permission enabling Graphite Bio to test the investigational, potentially revolutionary gene editing therapy GPH101 developed under the supervision of Matthew Porteus, MD, PhD, in a clinical trial for people with sickle cell disease (SCD). The California Institute for Regenerative … Continue reading CIRM-Funded Project Targeting Sickle Cell Disease Gets Green Light for Clinical Trial
Making a good thing better
Thomas Edison Legend has it that Thomas Edison “failed” 1,000 times before he managed to create the incandescent lightbulb. Edison says he didn’t get discouraged, instead he looked at each unsuccessful experiment as being one step closer to finding the method that really worked. That’s a lesson in optimism and persistence for all of us. … Continue reading Making a good thing better
UCLA scientists discover how SARS-CoV-2 causes multiple organ failure in mice
Heart muscle cells in an uninfected mouse (left) and a mouse infected with SARS-CoV-2 (right) with mitochondria seen in pink. The disorganization of the cells and mitochondria in the image at right is associated with irregular heartbeat and death. Image credit: UCLA Broad Stem Cell Center As the worldwide coronavirus pandemic rages on, scientists are trying … Continue reading UCLA scientists discover how SARS-CoV-2 causes multiple organ failure in mice
Positive results from CIRM-funded LAD-I trial presented at the 62nd American Society of Hematology Annual Meeting
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
You can’t take it if you don’t make it
Biomedical specialist Mamadou Dialio at work in the Cedars-Sinai Biomanufacturing Center. Photo by Cedars-Sinai. Following the race to develop a vaccine for COVID-19 has been a crash course in learning how complicated creating a new therapy is. It’s not just the science involved, but the logistics. Coming up with a vaccine that is both safe … Continue reading You can’t take it if you don’t make it
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
The Stem Cellar 