Knee joint pain rarely gets counted among major public‑health burdens. Yet it affects more than 3 million Californians and 34 million people across the U.S., with both massive economic and health consequences. Costly Impact In California alone, the cost of knee replacement surgeries exceeds $8 billion annually, according to Thomas Chalberg, PhD, founder and CEO … Continue reading Genascence discusses gene therapy for knee osteoarthritis
CIRM awarded Elpida Therapeutics a $3.9 million grant in December 2023 to support a pioneering preclinical study on Charcot Marie Tooth disease type 4J (CMT4J).
Dr. Noriyuki Kasahara from UCSF has been awarded $11.8 million by the California Institute for Regenerative Medicine (CIRM) to target newly diagnosed high-grade gliomas. This groundbreaking clinical trial aims to treat high-grade gliomas, one of the most challenging brain cancers to manage. These aggressive cancers develop in the brain or spinal cord and are notoriously … Continue reading Gene therapy trial tests cancer-killing virus in brain tumors
Regenerative medicine is a rapidly evolving field. As more therapies reach commercialization and clinical trial stages, there remains an immediate need to equip direct patient care nurses with skills to support patients on complex clinical trials as well as novel standard-of-care therapies. To address this need and continue efforts to build a regenerative medicine workforce … Continue reading Equipping California nurses with skills to support cell and gene therapy patients
Photo credit: UCSF Hataalii Tiisyatonii Begay (HT) is paving the road for newborns with SCID. When HT was born in 2018 in a remote part of the Navajo nation, he was quickly diagnosed with a rare and -usually fatal- condition. Today, thanks to a therapy developed at UCSF and funded by CIRM, he’s a healthy … Continue reading Update on SCID patient enrolled in CIRM-funded gene therapy trial
The California Institute for Regenerative Medicine (CIRM) is seeking applications for its next round of Quest Awards (DISC2) for discovery stage research. Applications are due August 2nd, 2022, at 2:00 PM PDT. Please visit the CIRM website for full details. The purpose of the Quest Awards is to promote the discovery of promising new stem cell-based or gene therapy … Continue reading Reminder! Apply now for discovery stage stem cell and gene therapy research funding
In October 2019, 20-year-old Jordan Janz became the first person in the world to receive an experimental therapy for cystinosis. Cystinosis is a rare genetic disorder characterized by the accumulation of an amino acid called cystine in different tissues and organs of the body including the kidneys, eyes, muscles, liver, pancreas, and brain. This accumulation … Continue reading An experimental gene therapy with a hairy twist
Until recently, “bespoke” meant one thing to me: a custom suit from London’s Savile Row. They’re beautiful—and expensive—so I assumed I’d never own anything bespoke. A tailored approach I was wrong. CIRM is now part of a bespoke effort far more important than clothing. This group aims to create tailor‑made gene therapies for rare diseases. … Continue reading Bespoke approach to rare diseases
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
Often on the Stem Cellar we feature CIRM-funded work that is helping advance the field, unlocking some of the secrets of stem cells and how best to use them to develop promising therapies. But every once in a while it’s good to remind ourselves that this work, while it may often seem slow, is already … Continue reading Celebrating a young life that almost wasn’t