Photo Courtesy of Andrea Fernandez de Soto Imagine a life where every time you step outside, everything in your environment poses a life-threatening risk. For children born with ADA-SCID (adenosine deaminase severe combined immunodeficiency), this is a reality. Their immune systems are so compromised that a common cold or a simple scrape on the knee … Continue reading Gene Therapy Brings Hope to Families Affected by Rare Immune Disorder
UCLA Broad Stem Cell Research Center
UCLA researchers exploring ways to prevent heart failure after heart attacks
ENPP1 is expressed in the region of scarring after injury to the heart. Cardiac muscle shown in red and ENPP1 in green demonstrates expression of ENPP1 in the region of scar formation. | Credit: Arjun Deb Lab/UCLA At the California Institute for Regenerative Medicine (CIRM), we support groundbreaking cardiovascular research from Discovery to Clinical as … Continue reading UCLA researchers exploring ways to prevent heart failure after heart attacks
How CIRM-funded research is bringing a lifesaving gene therapy to a 5-year-old
For her first year of life, Seersha Sulack stayed mostly in her bedroom because something as simple as a common cold could have killed her. The five-year-old was born with ADA-SCID, a condition so rare that only eight babies a year are born with it in the United States. Now, thanks to the work of … Continue reading How CIRM-funded research is bringing a lifesaving gene therapy to a 5-year-old
Bubble baby treatment cleared to restart clinical trial
Evie Vaccaro: Photo courtesy Nancy Ramos Three families battling a life-threatening immune disorder got some great news last week. A clinical trial that could save the life of their child has once again been given the go-ahead by the US Food and Drug Administration (FDA). The clinical trial is the work of UCLA’s Dr. Don … Continue reading Bubble baby treatment cleared to restart clinical trial
Developing a natural killer for cancer
Lili Yang UCLA Broad Stem Cell Research Center: Photo courtesy Reed Hutchinson PhotoGraphics When Lili Yang was studying for her PhD she approached her mentor, the Nobel Laureate Dr. David Baltimore, and told him she was thinking about writing her thesis on a combination of gene therapy, immunotherapy and stem cell therapy. She says he … Continue reading Developing a natural killer for cancer
Join us to hear how stem cell and gene therapy are taking on diseases of aging
It is estimated that as many as 90 percent of people in industrialized countries who die every day, die from diseases of aging such as heart disease, stroke, and cancer. Of those still alive the numbers aren’t much more reassuring. More than 80 percent of people over the age of 65 have a chronic medical … Continue reading Join us to hear how stem cell and gene therapy are taking on diseases of aging
Chance discovery could lead to a treatment for skin ulcers
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Dr. Antoni Ribas in his research lab on the UCLA Campus: Photo courtesy Ann Johansson When UCLA's Dr. Antoni Ribas was researching a potential therapy for melanoma, a form of skin cancer, he stumbled upon something unexpected. That unexpected discovery has now resulted in him getting … Continue reading Chance discovery could lead to a treatment for skin ulcers
How two California researchers are advancing world class science to develop real life solutions
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST In our recently launched 5-year Strategic Plan, the California Institute for Regenerative Medicine (CIRM) profiled two researchers who have leveraged CIRM funding to translate basic biological discoveries into potential real-world solutions for devastating diseases. Dr. Joseph Wu is director of the Stanford Cardiovascular Institute and the recipient … Continue reading How two California researchers are advancing world class science to develop real life solutions
UCLA gene therapy offers children with LAD-1 a new chance at living a normal life
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Photo courtesy of Tamara Hogue/UCLA Broad Stem Cell Research Center Leukocyte adhesion deficiency type 1 (LAD-1) is a rare pediatric disorder that causes the immune system to malfunction, resulting in recurrent, often severe, bacterial and fungal infections as well as delayed wound healing. This is because of a missing protein that would normally … Continue reading UCLA gene therapy offers children with LAD-1 a new chance at living a normal life
One step closer to making ‘off-the-shelf’ immune cell therapy for cancer a reality
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Immunotherapy is a type of cancer treatment that uses a person's own immune system to fight cancer. It comes in a variety of forms including targeted antibodies, cancer vaccines, and adoptive cell therapies. While immunotherapies have revolutionized the treatment of aggressive cancers in recent decades, they must be created … Continue reading One step closer to making ‘off-the-shelf’ immune cell therapy for cancer a reality
The Stem Cellar 