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
Inaugural UC San Diego Gene Therapy Initiative Symposium is fueling progress in the field
There was no shortage of enthusiasm at the inaugural UC San Diego Gene Therapy Initiative Symposium hosted at the Roth Auditorium at the prestigious Sanford Consortium, where researchers, clinicians, academia, industry, and patient advocates shared discoveries in gene therapy, a modern-day treatment that’s revolutionizing medicine.CIRM was a platinum sponsor of the event.The gene therapy field … Continue reading Inaugural UC San Diego Gene Therapy Initiative Symposium is 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 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.
Reasons to be thankful this Thanksgiving: creative nerds
We at the California Institute for Regenerative Medicine have a lot to be thankful for this Thanksgiving. We get to work with some extraordinary colleagues, we get to know some remarkable patient advocates who are pioneers in volunteering for stem cell and gene therapies, and we have a front row seat in a movement that … Continue reading Reasons to be thankful this Thanksgiving: creative nerds
How the Tooth Fairy is helping unlock the secrets of autism
Our 2021-22 Annual Report is now online. It’s filled with information about the work we have done over the last year (we are on a fiscal calendar year from July 1 – June 30), the people who have helped us do that work, and some of the people who have benefited from that work. One … Continue reading How the Tooth Fairy is helping unlock the secrets of autism
An experimental gene therapy with a hairy twist
In October 2019, 20-year-old Jordan Janz became the first person in the world to receive an experimental therapy for cystinosis. Cystinosis is a rare genetic disorder characterized by the accumulation of an amino acid called cystine in different tissues and organs of the body including the kidneys, eyes, muscles, liver, pancreas, and brain. This accumulation … Continue reading An experimental gene therapy with a hairy twist
Meet the man who is unlocking the secrets of autism and sending mini-brains into space
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Dr. Alysson Muotri, UC San Diego Normally if you meet someone who has a mini-fridge filled with brains, your first thought is to call the police. But when that someone is Dr. Alysson Muotri, a professor at U.C. San Diego, your second thought is “do tell … Continue reading Meet the man who is unlocking the secrets of autism and sending mini-brains into space
Stem Cell Agency Board Invests in Therapy Targeting Deadly Blood Cancers
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Dr. Ezra Cohen, photo courtesy UCSD Hematologic malignancies are cancers that affect the blood, bone marrow and lymph nodes and include different forms of leukemia and lymphoma. Current treatments can be effective, but in those patients that do not respond, there are few treatment options. Today, … Continue reading Stem Cell Agency Board Invests in Therapy Targeting Deadly Blood Cancers
Retooling a COVID drug to boost its effectiveness
Coronavirus particles, illustration. When the COVID-19 pandemic broke out scientists scrambled to find existing medications that might help counter the life-threatening elements of the virus. One of the first medications that showed real promise was remdesivir. It’s an anti-viral drug that was originally developed to target novel, emerging viruses, viruses like COVID19. It was approved … Continue reading Retooling a COVID drug to boost its effectiveness
The Stem Cellar 