Gene therapy is life-changing for children with a life-threatening brain disorder

If you have never heard of AADC deficiency count yourself lucky. It’s a rare, incurable condition that affects only around 135 children worldwide but it’s impact on those children and their families is devastating. The children can’t speak, can’t feed themselves or hold up their head, they have severe mood swings and often suffer from … Continue reading Gene therapy is life-changing for children with a life-threatening brain disorder

Stem cell treatment improves motor function in monkeys modeling Parkinson’s Disease

Neurodegenerative diseases impact millions of people worldwide with the risk of being affected by one of these diseases increasing as you get older. For many of these diseases, there are very few treatments available to patients. As life expectancy increases and the population continues to age, it is crucial to try and find treatments that … Continue reading Stem cell treatment improves motor function in monkeys modeling Parkinson’s Disease

A new way to evade immune rejection in transplanting cells

Immune fluorescence of HIP cardiomyocytes in a dish; Photo courtesy of UCSF Transplanting cells or an entire organ from one person to another can be lifesaving but it comes with a cost. To avoid the recipient’s body rejecting the cells or organ the patient has to be given powerful immunosuppressive medications. Those medications weaken the … Continue reading A new way to evade immune rejection in transplanting cells

Hollywood and Patient Advocacy – two people who are on our Board but never boring

At first glance, Lauren Miller Rogen and David Higgins, PhD, seem an unlikely pair. She’s an actor, writer, and director and has worked with some of the biggest names in Hollywood. He has a doctorate in molecular biology and genetics and has worked at some of the most well-known companies in biotech. But together they … Continue reading Hollywood and Patient Advocacy – two people who are on our Board but never boring

We’ve got cash, here’s how you can get some

When the voters of California approved Proposition 14 last November (thanks folks) they gave us $5.5 billion to continue the work we started way back in 2014. It’s a great honor, and a great responsibility. It’s also a great opportunity to look at what we do and how we do it and try to come … Continue reading We’ve got cash, here’s how you can get some

Heads or tails? Stem cells help guide the decision

Two cell embryo There are many unknown elements for what triggers the cells in an embryo to start dividing and multiplying and becoming every single cell in the body. Now researchers at the Gladstone Institutes in San Francisco have uncovered one of those elements, how embryos determine which cells become the head and which the … Continue reading Heads or tails? Stem cells help guide the decision

CIRM funded trial for AMD shows promising results

This upcoming July is healthy vision month, a time to remember the importance of making vision and eye health a priority. It's also a time to think about the approximately 12 million people, 40 and over in the United States, that have a vision impairment. Vision can be something that many of us take for … Continue reading CIRM funded trial for AMD shows promising results

Welcoming back old friends and some new ones

When Proposition 14 was approved by voters in November we were given a chance to carry on the work we have been doing for more than 16 years. What we hadn't anticipated was that we would also get a chance to do that with some of the team that helped us make CIRM what it … Continue reading Welcoming back old friends and some new ones

Physicians and patient advocates on the front lines of the fight for a more equitable health system

Over the last year there has been increasing awareness of the inequalities in the American healthcare system. At every level there is evidence of bias, discrimination and unequal access to the best care. Sometimes unequal access to any care. That is, hopefully, changing but only if the new awareness is matched with action. At the … Continue reading Physicians and patient advocates on the front lines of the fight for a more equitable health system

CIRM Board Approves New Clinical Trial for ALS

This past Friday the governing Board of the California Institute for Regenerative Medicine (CIRM) awarded $11.99 million to Cedars-Sinai to fund a clinical trial for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.  ALS is a neurodegenerative disease that results in the death of nerve cells in the brain and spinal cord, causing … Continue reading CIRM Board Approves New Clinical Trial for ALS