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CIRM funded trial may pave way for gene therapy to treat different diseases
Image Description: Jordan Janz (left) and Dr. Stephanie Cherqui (right) According to the National Organization for Rare Disorders (NORD), a disease is consider rare if it affects fewer than 200,000...
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Celebrating a young life that almost wasn’t
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...
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UCLA gene therapy offers children with LAD-1 a new chance at living a normal...
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...
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Creating a ‘bespoke’ approach to rare diseases
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Up until recently the word “bespoke” meant just one thing to me, a hand-made suit, customized and fitted to you. There’s a street in London, Saville Row,...
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An experimental gene therapy with a hairy twist
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...
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Reminder! Apply now for discovery stage stem cell and gene therapy research...
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...
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Update on SCID patient enrolled in CIRM-funded gene therapy trial
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...
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Equipping California nurses with skills to support cell and gene therapy...
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...
View ArticleGene therapy trial tests cancer-killing virus in brain tumors
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...
View ArticleInaugural UC San Diego Gene Therapy Initiative Symposium is fueling progress...
There was no shortage of enthusiasm at the inaugural UC San Diego Gene Therapy Initiative Symposium hosted at the Roth Auditorium at the prestigious Sanford Consortium, where researchers, clinicians,...
View ArticleStory of Hope: Elpida Therapeutics’ Pioneer Study on Rare Nerve Disease CMT4J
Image source: SickKids -Terry, Georgia, and Michael Pirovolakis CIRM awarded Elpida Therapeutics a $3.9 million grant in December 2023 to support a pioneering preclinical study on Charcot Marie Tooth...
View ArticleCIRM Showcases Strategic Vision and New Initiatives at 7th Annual Alpha...
Leveraging CIRM Infrastructure to Improve Accessibility Panel - From left to right: Noah Federman, MD (moderator), Catriona Jamieson, MD, PhD, Mehrdad Abedi, MD, Daniela Bota, MD, PhD, Thomas Buchanan,...
View ArticleAdvancing Heart Health: CIRM Funds Tenaya Therapeutics’ Gene Therapy Trial...
Histology of human cardiac muscle under microscope. Credit: Shutterstock The California Institute for Regenerative Medicine (CIRM) recently announced funding a series of projects spanning the earliest...
View ArticleCIRM awards $2.8 million to develop a gene therapy for rare neuromuscular...
Credit: Shutterstock The California Institute for Regenerative Medicine (CIRM) announced in January $100 million in funding for a broad range of projects, including four to support clinical programs....
View ArticleGene Therapy for Osteoarthritis Shows Positive Results in Early Trial
Image Credit: Shutterstock Here’s a shocking statistic: in 2020, 7.6% of the world’s population was living with the most common form of arthritis – osteoarthritis (OA). That amounts to 595 million...
View ArticleGene Therapy for Heart Failure: A Conversation with Eric Adler, MD
Image Credit: The Adler Lab at the University of California, San Diego In the fall of 2024, the California Institute for Regenerative Medicine (CIRM) helped sponsor the Gene Therapy Initiative...
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