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
gene therapy
Funding a Clinical Trial for a Functional Cure for HIV
Antiretroviral drugs have turned HIV/AIDS from a fatal disease into one that can often be controlled, but they are not a cure. That’s why the CIRM Board approved $6.85 million for a therapy designed to cure the disease. This is CIRM’s 82nd funded clinical trial. Millions impacted About 38 million people worldwide live with HIV/AIDS, … Continue reading Funding a Clinical Trial for a Functional Cure for HIV
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
Reminder! Apply now for discovery stage stem cell and gene therapy research funding
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
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
Bespoke approach to rare diseases
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
How do Zebrafish grow ears? It’s quite transparent
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Zebrafish One of the hopes of regenerative medicine is that one day we will be able to use stem cells to regrow damaged organs, avoiding the need for a transplant. It’s a fascinating idea, supported in part by the ability of some creatures, such as Axolotls … Continue reading How do Zebrafish grow ears? It’s quite transparent
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
A personal reason to develop a better gene therapy
THIS BLOG IS ALSO AVAILABLE AS AN AUDIOCAST ON SPOTIFY Credit : Allison Dougherty, Broad Communications For Sharif Tabebordbar, finding a gene therapy for genetic muscle wasting diseases was personal. When he was a teenager, his father was diagnosed with a rare genetic muscle disease that eventually left him unable to walk. In an interview … Continue reading A personal reason to develop a better gene therapy
Tiny tools for the smallest of tasks, editing genes
YOU CAN LISTEN TO THIS BLOG AS AN AUDIOCAST ON SPOTIFY Developing new tools to edit genes Having the right tools to do a job is important. Try using a large screwdriver to tighten the screws on your glasses and you quickly appreciate that it’s not just the type of tool that’s important, it’s also … Continue reading Tiny tools for the smallest of tasks, editing genes