CIRM-funding supports world’s first stem cell treatment for spina bifida delivered during fetal surgery

Dr. Diana Farmer (right) meets with Emily Lofton and her baby Robbie who had stem cell treatment for spina bifida in the womb. Photo: UC Davis Health The California Institute for Regenerative Medicine (CIRM) recently shared some encouraging news on The Stem Cellar about a CIRM-funded stem cell clinical trial for spina bifida at UC … Continue reading CIRM-funding supports world’s first stem cell treatment for spina bifida delivered during fetal surgery

Strength forged from adversity

Regina Karchner - Photo courtesy Nancy Ramos Photography 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 … Continue reading Strength forged from adversity

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

So far, some encouraging news for stem cell clinical trial treating epilepsy

Neurona Therapeutics is testing a new therapy for a drug-resistant form of epilepsy and has just released some encouraging early findings. The first patient treated went from having more than 30 seizures a month to just four seizures over a three-month period. This clinical trial, funded by the California Institute for Regenerative Medicine (CIRM), is … Continue reading So far, some encouraging news for stem cell clinical trial treating epilepsy

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

CIRM-funded stem cell-gene therapy shows promise in ALS safety trial

Senior author of the study Clive Svendsen, PhD (center) With funding support from the California Institute for Regenerative Medicine (CIRM), Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. This combined stem cell and gene therapy can potentially protect diseased motor neurons … Continue reading CIRM-funded stem cell-gene therapy shows promise in ALS safety trial

How stem cells helped Veronica fight retinitis pigmentosa and regain her vision

Veronica and Elliott Growing up Veronica McDougall thought everyone saw the world the way she did; blurry, slightly out-of-focus and with tunnel vision.  As she got older her sight got worse and even the strongest prescription glasses didn’t help. When she was 15 her brother tried teaching her to drive. One night she got into … Continue reading How stem cells helped Veronica fight retinitis pigmentosa and regain her vision

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