Image Credit: The Adler Lab at the University of California, San Diego In the fall of 2024, the California Institute for Regenerative Medicine (CIRM) helped sponsor the Gene Therapy Initiative symposium in La Jolla, CA. While there, CIRM staff caught up with a few CIRM grantees who are working on gene therapy approaches to treating … Continue reading Gene Therapy for Heart Failure: A Conversation with Eric Adler, MD
UC San Diego
CIRM Funds new UCSD VERGE Center to Better Understand Schizophrenia and Autism
From top left to right: Anne Bang, PhD; Carrie Bearden, PhD; Eugene Yeo, PhD; Jonathan Sebat, PhD Bottom left to right: Alysson Moutri, PhD; Lilia Iakoucheva, PhD; Sébastien Jacquemont; Trey Ideker, PhD A team of UC San Diego researchers with support of a $12.5 million grant from the California Institute for Regenerative Medicine (CIRM) plans … Continue reading CIRM Funds new UCSD VERGE Center to Better Understand Schizophrenia and Autism
UC San Diego Gene Therapy Initiative Symposium fueling progress in the field
Enthusiasm ran high at the inaugural UC San Diego Gene Therapy Initiative Symposium at the Roth Auditorium at the Sanford Consortium. Researchers, clinicians, and patient advocates gathered to share new discoveries in gene therapy—a rapidly evolving field that is reshaping modern medicine. CIRM served as a platinum sponsor of the event. Era of progress The … Continue reading UC San Diego Gene Therapy Initiative Symposium fueling progress in the field
Novartis acquisition validates CIRM’s impact on cystinosis treatment
Dr. Stephanie Cherqui The future looks brighter for cystinosis patients and their families. As one of 7,000 rare diseases, cystinosis causes an abnormal build-up of the amino acid cystine that can lead to organ failure and premature death. With little to no treatment options, children and young adults primarily affected by the condition often have … Continue reading Novartis acquisition validates CIRM’s impact on cystinosis treatment
Making kidney 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 bodies reject the transplant. Improving kidney transplant outcomes 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 … Continue reading Making kidney transplants easier for kids and charting a new approach to fighting solid tumors.
Unlocking the secrets of autism
Dr. Alysson Muotri, in his lab at UCSD For Dr. Alysson Muotri, unlocking autism’s biology is personal. His son has autism, and Muotri hopes to help him and millions of others. A Mission to Understand Autism He launched the Tooth Fairy Project, which collected more than 3,000 donated baby teeth from children with and without … Continue reading Unlocking the secrets of autism
An experimental cystinosis treatment changed hair color
In October 2019, 20‑year‑old Jordan Janz became the first person to receive an experimental gene therapy for cystinosis. A rare disorder Cystinosis is a rare genetic disorder in which cystine builds up in organs such as the kidneys, eyes, muscles, liver, pancreas, and brain. This buildup leads to multi‑organ failure and usually causes premature death. … Continue reading An experimental cystinosis treatment changed hair color
Studying autism using brain organoids
Dr. Alysson Muotri, UC San Diego Normally, if someone keeps a mini‑fridge full of brains, your first instinct is to call the police. But when that person is Dr. Alysson Muotri of UC San Diego, your next thought is, “Tell me more.” Muotri is fascinated by the human brain. His research explores how the brain … Continue reading Studying autism using brain organoids
CIRM Funds Development of New CAR T-Cell Therapy for Blood Cancers
Dr. Ezra Cohen, photo courtesy UCSD Patients with blood cancers often respond well to existing treatments. However, those therapies do not work for everyone. For patients whose disease resists standard treatment, options can be limited. To address that need, the California Institute for Regenerative Medicine (CIRM) has awarded $4.1 million to support the development of … Continue reading CIRM Funds Development of New CAR T-Cell Therapy for Blood Cancers
Researchers develop a promising new version of Remdesivir
Coronavirus particles, illustration When COVID-19 emerged, scientists worldwide raced to identify treatments that could reduce severe illness and save lives. One of the first antiviral drugs to show real promise was remdesivir. The FDA approved the treatment in October 2020 after studies showed it could help some hospitalized patients recover more quickly. However, remdesivir has … Continue reading Researchers develop a promising new version of Remdesivir