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
bioengineering
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
Stem Cell Agency Expands Industry Alliance Program to Accelerate Therapies
An ever-growing array of academic and industry resources are required to rapidly translate scientific discoveries and emerging technologies toward safe and effective regenerative medicine therapies for patients. To help, the California Institute for Regenerative Medicine (CIRM) is creating a network of Industry Resource Partners (IRP) that will make its unique resources available to help accelerate … Continue reading Stem Cell Agency Expands Industry Alliance Program to Accelerate Therapies
Fighting for his life and the lives of other stroke survivors
Sean Entin, stroke survivor and founder of Stroke Hacker The word "miraculous" gets tossed around a lot in the world of medicine, mostly by people who have made an unexpected recovery from a deadly or life-threatening condition. In Sean Entin's case calling his recovery from an almost-fatal stroke could be called miraculous, but I think … Continue reading Fighting for his life and the lives of other stroke survivors
The present and future of regenerative medicine
One of the great pleasures of my job is getting to meet the high school students who take part in our SPARK or Summer Internship to Accelerate Regenerative Medicine Knowledge program. It's a summer internship for high school students where they get to spend a couple of months working in a world class stem cell … Continue reading The present and future of regenerative medicine
Using stem cells and smart machines to warn of heart problems
Despite advances in treatments in recent years heart disease remains the leading cause of death in the US. It accounts for one in three deaths in this country, and many people are not even aware they have a problem until they have a heart attack. One of the early warning signs of danger is a … Continue reading Using stem cells and smart machines to warn of heart problems
A big deal for type 1 diabetes
It’s not often you get excited talking about company mergers, but a deal announced today is something worth getting excited about, particularly if you have type 1 diabetes (T1D). Today Vertex announced it was buying ViaCyte for $320 million in cash. Why is that important? Because both companies are working on developing stem cell therapies … Continue reading A big deal for type 1 diabetes
First patient dosed in clinical trial for a drug-resistant form of epilepsy
Tablet BM47753. Neo-Babylonian Period. Courtesy of the British Museum, London. Epilepsy seems to have been a problem for people for as long as people have been around. The first recorded mention of it is on a 4000-year-old Akkadian tablet found in Mesopotamia (modern day Iraq). The tablet includes a description of a person with "his … Continue reading First patient dosed in clinical trial for a drug-resistant form of epilepsy
Stem Cell Agency Board Invests in 19 Discovery Research Programs Targeting Cancers, Heart Disease and Other Disorders
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Dr. Judy Shizuru, Stanford University While stem cell and gene therapy research has advanced dramatically in recent years, there are still many unknowns and many questions remaining about how best to use these approaches in developing therapies. That’s why the governing Board of the California Institute … Continue reading Stem Cell Agency Board Invests in 19 Discovery Research Programs Targeting Cancers, Heart Disease and Other Disorders
The Stem Cellar 