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
gene therapy
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 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
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, that specializes in these suits. They’re gorgeous. They’re also very expensive and so I thought I’d never have a … Continue reading Creating a ‘bespoke’ approach to rare diseases
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
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 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
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 people. If you combine the over 7,000 known rare diseases, about 30 million people in the U.S. are affected by one of these conditions. A … Continue reading CIRM funded trial may pave way for gene therapy to treat different diseases
CIRM-Funded Clinical Trial for Sickle Cell Gives Hope to People Battling the Disease
Marissa Cors (right) with her mother Adrienne Shapiro Marissa Cors has lived with Sickle Cell Disease (SCD) for more than 40 years. The co-founder of The Sickle Cell Experience Live, an online platform designed to bring more awareness to Sickle Cell Disease around the world, says it’s hard, knowing that at any moment you may … Continue reading CIRM-Funded Clinical Trial for Sickle Cell Gives Hope to People Battling the Disease
Scientists Engineer Stem Cells to Fight HIV
Image of the virus that causes AIDS - courtesy NIH If that headline seems familiar it should. It came from an article in MIT Technology Review back in 2009. There have been many other headlines since then, all on the same subject, and yet here we are, in 2020, and still no cure for HIV/AIDS. … Continue reading Scientists Engineer Stem Cells to Fight HIV
‘A Tornado at the Front Door, a Tsunami at the Back Door’
CIRM funds a lot of research and all of it has life-saving potential. But every once in a while you come across a story about someone benefiting from CIRM-supported research that highlights why the work we do is so important. This story is about a brilliant researcher at UC San Diego developing a treatment for … Continue reading ‘A Tornado at the Front Door, a Tsunami at the Back Door’
NIH collaboration aims to develop affordable gene therapies for sickle cell disease and HIV
Sickle cell disease (SCD) and HIV have a major burden on the health of impoverished communities all over the world. Of the 38 million people living with HIV all over the world, approximately 95% reside within developing countries, with 67% in sub-Saharan Africa, half of whom are living without any treatment. Fifteen million babies will … Continue reading NIH collaboration aims to develop affordable gene therapies for sickle cell disease and HIV
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