Stock image of the Trisomy 21 chromosome The California Institute for Regenerative Medicine (CIRM) recently awarded $1.5 million to Denise Al Alam, PhD, of the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center to support research that aims to understand lung disease in individuals with Trisomy 21, also known as Down Syndrome.Although Trisomy 21 … Continue reading CIRM awards $1.5 million in lung health study for patients with Down Syndrome
Genetic Disorders
Making transplants easier for kids, and charting a new approach to fighting solid tumors.
Every year California performs around 100 kidney transplants in children but, on average, around 50 of these patients will have their body reject the transplant. These children then have to undergo regular dialysis while waiting for a new organ. Even the successful transplants require a lifetime of immunosuppression medications. These medications can prevent rejection but … Continue reading Making transplants easier for kids, and charting a new approach to fighting solid tumors.
Stem Cell Agency Board Invests in 19 Discovery Research Programs Targeting Cancers, Heart Disease and Other Disorders
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Dr. Judy Shizuru, Stanford University While stem cell and gene therapy research has advanced dramatically in recent years, there are still many unknowns and many questions remaining about how best to use these approaches in developing therapies. That’s why the governing Board of the California Institute … Continue reading Stem Cell Agency Board Invests in 19 Discovery Research Programs Targeting Cancers, Heart Disease and Other Disorders
HOPE for patients with a muscle destroying disease
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Caleb Sizemore, photo by Todd Dubnicoff Caleb Sizemore says growing up with Duchenne’s Muscular Dystrophy (DMD) was tough. The disease is a rare genetic disorder that slowly destroys a person’s muscles, impairing their ability to walk or breathe. Eventually it attacks the heart leading to premature … Continue reading HOPE for patients with a muscle destroying disease
Joining the movement to fight rare diseases
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST It’s hard to think of something as being rare when it affects up to 30 million Americans and 300 million people worldwide. But the truth is there are more than 6,000 conditions – those affecting 200,000 people or fewer – that are considered rare. Today, … Continue reading Joining the movement to fight rare diseases
Stem cell gene therapy for Fabry disease shows positive results in patients
Darren Bidulka rests after his modified blood stem cells were transplanted into him at the Foothills Medical Centre in Calgary in 2017, allowing him to stop his enzyme therapy. (From left): Dr. Jeffrey Medin, Medical College of Wisconsin, Dr. Aneal Khan, the experimental trial lead in Calgary, and Darren Bidulka. Image Credit: Darren Bidulka Fabry … Continue reading Stem cell gene therapy for Fabry disease shows positive results in patients
Biotechnology companies join forces in developing treatment for X-SCID
Jasper Therapeutics, Inc., a biotechnology company focused on blood stem cell therapies, and Graphite Bio, Inc., a biotechnology company focused on gene editing therapies to treat or cure serious diseases, announced a research and clinical collaboration for a treatment for X-SCID. X-SCID, which stands for X-linked severe combined immunodeficiency, is a genetic disorder that interferes … Continue reading Biotechnology companies join forces in developing treatment for X-SCID
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
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
CIRM Board Approves Four New Clinical Trials
A breakdown of CIRM's clinical trials by disease area This past Thursday the governing Board of the California Institute for Regenerative Medicine (CIRM) approved four new clinical trials in addition to ten new discovery research awards. These new awards bring the total number of CIRM-funded clinical trials to 68. Additionally, these new additions have allowed … Continue reading CIRM Board Approves Four New Clinical Trials