Top left to right: Tomasz Nowakowski, PhD, and Nevan Krogan, PhD Bottom left to right: Aparna Bhaduri, PhD; Matt State, PhD; Alice Ting, PhD; Trey Ideker, PhD; Willow Coyote-Maestas, PhD As outlined in Proposition 14, one of the goals of the California Institute for Regenerative Medicine (CIRM) is to help accelerate discoveries that enhance our … Continue reading Using Genetic Sequencing and AI to Identify and Treat Schizophrenia
Biotechnology
Turning Hope into Reality: How a Genetic Therapy Helped Connor
Photo credit: n-Lorem Connor Dalby is a 15-year-old teenager who loves music and the beach, but his journey has been far from ordinary. Diagnosed with a rare mutation in the SCN2A gene, his life has been filled with challenges. From the time he was just a few weeks old, he had trouble sleeping and struggled … Continue reading Turning Hope into Reality: How a Genetic Therapy Helped Connor
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
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
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
Funding a Clinical Trial for a Functional Cure for HIV
The use of antiretroviral drugs has turned HIV/AIDS from a fatal disease to one that can, in many cases in the US, be controlled. But these drugs are not a cure. That's why the governing Board of the California Institute for Regenerative Medicine (CIRM) voted to approve investing $6.85 million in a therapy that aims … Continue reading Funding a Clinical Trial for a Functional Cure for HIV
Fast Track Designation for a therapy making transplants safer for children with a fatal immune disorder
Bone marrow transplant For children born with severe combined immunodeficiency (SCID) life can be very challenging. SCID means they have no functioning immune system, so even a simple infection can prove life threatening. Left untreated, children with SCID often die in the first few years of life. There are stem cell/gene therapies funded by the … Continue reading Fast Track Designation for a therapy making transplants safer for children with a fatal immune disorder
The race to cure sickle cell disease
September is National Sickle Cell Awareness Month, a time to refocus our efforts to find new treatments, even a cure, for people with sickle cell disease. Until we get those, CIRM remains committed to doing everything we can to reduce the stigma and bias that surrounds it. Sickle cell disease (SCD) is a rare, inherited … Continue reading The race to cure sickle cell disease
A better, faster, more effective way to edit genes
Clinical fellow Brian Shy talks with postdoctoral scholar Tori Yamamoto in the Marson Lab at Gladstone Institutes on June 8th, 2022. Photo courtesy Gladstone Institutes. For years scientists have been touting the potential of CRISPR, a gene editing tool that allows you to target a specific mutation and either cut it out or replace it … Continue reading A better, faster, more effective way to edit genes
The Stem Cellar 