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
UC San Diego
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.
CIRM Board Approves Funding for New Clinical Trial Targeting Brain Tumors
The governing Board of the California Institute for Regenerative Medicine (CIRM) has awarded almost $12 million to carry out a clinical trial targeting brain tumors. This brings the total number of CIRM funded clinical trials to 83. $11,999,984 was awarded to Dr. Jana Portnow at the Beckman Research Institute of City of Hope. They are using Neural stem … Continue reading CIRM Board Approves Funding for New Clinical Trial Targeting Brain 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
From our house to the White House. Kinda
Jackie Ward, PhD. Photo courtesy National Institute of Neurological Diseases and Stroke It's always fun to meet someone early in their career and see how they grow and evolve and take on new challenges. I first met Jackie Ward when she received a training grant from CIRM while she studied for her PhD at the … Continue reading From our house to the White House. Kinda
How CIRM contributed to City of Hope study helping man with HIV into long-term remission
The news that a stem cell transplant at City of Hope helped a man with HIV go into long-term remission made banner headlines around the world. As it should. It’s a huge achievement, particularly as the 66-year-old man had been living with HIV since 1988. What wasn’t reported was that work supported by the California … Continue reading How CIRM contributed to City of Hope study helping man with HIV into long-term remission
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
CIRM Board gives thumbs up to training and treatment programs
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST CIRM Bridges student discusses her poster presentation At CIRM, the bread and butter of what we do is funding research and hopefully advancing therapies to patients. But the jam, that's our education programs. Helping train the next generation of stem cell and gene therapy scientists is … Continue reading CIRM Board gives thumbs up to training and treatment programs
Two Early-Stage Research Programs Targeting Cartilage Damage Get Funding from Stem Cell Agency
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Darryl D'Lima: Scripps Health Every year millions of Americans suffer damage to their cartilage, either in their knee or other joints, that can eventually lead to osteoarthritis, pain and immobility. Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved two projects targeting … Continue reading Two Early-Stage Research Programs Targeting Cartilage Damage Get Funding from Stem Cell Agency