Image Source: American Heart Association Journals The California Institute for Regenerative Medicine (CIRM) recently awarded $4 million to support the development of a one-time gene therapy for desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM), a rare and life-threatening genetic disorder. This preclinical award to Deborah D. Ascheim, MD, and her team at Rejuvenate Bio are … Continue reading CIRM $4 million awarded to support innovative Gene Therapy for Rare Heart Disorder
Genetic Disorders
CIRM awards $1.5 million in lung health study for patients with Down Syndrome
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
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
Celebrating National DNA Day Together
DNA provides the code of life for nearly all living organisms. So, it’s no wonder that scientists have been studying DNA and the human genome (complete set of DNA) for decades. In April 1953, James Watson and Francis Crick, in collaboration with Rosalind Franklin, first described the structure of DNA as a double helix. In … Continue reading Celebrating National DNA Day Together
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
Looking back and looking forward: good news for two CIRM-supported studies
https://open.spotify.com/episode/4IimCtQ8STpFikwZRXqSAB Dr. Rosa Bacchetta on the right with Brian Lookofsky (left) and Taylor Lookofsky after CIRM funded Dr. Bacchetta's work in October 2019. Taylor has IPEX syndrome It’s always lovely to end the week on a bright note and that’s certainly the case this week, thanks to some encouraging news about CIRM-funded research targeting blood … Continue reading Looking back and looking forward: good news for two CIRM-supported studies
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
The Stem Cellar 