Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment

A 12 year-old Kurt Gillenberg. Photo courtesy of Kurt Gillenberg. When Kurt Gillenberg was 10 months old, his parents knew something wasn’t right. But it wasn’t until he reached 18 months that they found a doctor at the University of California, San Diego, who diagnosed Kurt with cystinosis – a rare genetic, metabolic defect that … Continue reading Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment

Stem Cell Awareness: What are stem cells and why research is important 

Have you heard of stem cells but don't know what they are or why they are essential in medical research? In celebration of Stem Cell Awareness Day, the second Wednesday in October that is dedicated to educating about the importance of stem cells, the California Institute for Regenerative Medicine (CIRM) is providing this guide on … Continue reading Stem Cell Awareness: What are stem cells and why research is important 

CIRM invests $2.7 million in research to develop stem cell-based tendon tissue for shoulder injuries

The California Institute for Regenerative Medicine (CIRM) has awarded a $2.7 million research grant to Scripps Health to investigate the use of stem cells to engineer rotator cuff tendons in the lab that can then be used to repair common shoulder injuries.   The rotator cuff is a group of four muscles and their attached tendons, … Continue reading CIRM invests $2.7 million in research to develop stem cell-based tendon tissue for shoulder injuries

Finding a treatment for Tay-Sachs disease

The California Institute for Regenerative Medicine (CIRM) has awarded $4,048,253 to Dr. Joseph Anderson and his team at UC Davis to develop a blood stem cell gene therapy for the treatment of Tay-Sachs disease.   Tay-Sachs disease is a rare genetic disorder where a deficiency in the Hex A gene results in excessive accumulation of … Continue reading Finding a treatment for Tay-Sachs disease

Lab-made retinas offer a new approach to battling vision loss

Age-related macular degeneration (AMD) is the leading cause of vision loss in the elderly. Now, new research using 3D organoid models of the eye has uncovered clues as to what happens in AMD, and how to stop it.  In AMD, a person loses their central vision because the light sensitive cells in the macula, a … Continue reading Lab-made retinas offer a new approach to battling vision loss

High school SPARK intern presents stem cell research to academic audience 

Earlier this year, CIRM welcomed many energetic and enthusiastic high school students at the 2022 SPARK Program annual conference in Oakland. The SPARK program is one of the California Institute for Regenerative Medicine’s (CIRM) many programs dedicated to building a diverse and highly-skilled workforce to support the growing regenerative medicine economy right here in California.    … Continue reading High school SPARK intern presents stem cell research to academic audience 

Tratando malformaciones congénitas antes del nacimiento 

El bebé, Tobi recibió un tratamiento de células madre, financiado por el CIRM, mientras aún estaba en el útero. To read this blog in English, click here. Michelle y Jeff se llenaron de felicidad cuando se enteraron de que iban a tener un bebé.   Luego, un examen de ultrasonido a las 20 semanas del embarazo … Continue reading Tratando malformaciones congénitas antes del nacimiento 

Study could pave the way in reducing decline in muscle strength as people age 

A study by Stanford Medicine researchers in older mice may lead to treatments that help seniors regain muscle strength lost to aging. Muscle stem cells—which are activated in response to muscle injury to regenerate damaged muscle tissue—lose their potency with age. A study from the National Health and Nutrition Examination Survey showed that five percent … Continue reading Study could pave the way in reducing decline in muscle strength as people age 

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

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