The California Institute for Regenerative Medicine (CIRM) awarded $5,444,353 to Dr. Natalia Gomez-Ospina and her team at Stanford University for a late-stage preclinical program targeting Severe Mucopolysaccharidosis type 1, also known as Hurler syndrome. This inherited condition is caused by a faulty gene. Children with Hurler syndrome lack an enzyme that the body needs to digest sugar. … Continue reading Investing in a stem cell treatment for Hurler syndrome
gene therapy
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 -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
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
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
Zebrafish One hope 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. Learning from other species It’s a fascinating idea, supported in part by the ability of some creatures, such as Axolotls and salamanders, to regrow parts of their … Continue reading How do Zebrafish grow ears? It’s quite transparent
UCLA gene therapy for LAD-1
Photo courtesy of Tamara Hogue/UCLA Broad Stem Cell Research Center Leukocyte adhesion deficiency type 1 (LAD‑1) is a rare pediatric disorder that cripples the immune system. Children experience recurrent, severe bacterial and fungal infections and struggle to heal wounds. The disease results from a missing protein that normally allows white blood cells to leave blood … Continue reading UCLA gene therapy for LAD-1
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
Celebrating Ja’Ceon Golden
Meet Ja’Ceon Golden. He was among the first patients treated at UC San Francisco, in partnership with St. Jude Children’s Research Hospital, in a CIRM-funded study for Severe Combined Immunodeficiency. Ja’Ceon was born without a functioning immune system, so even a simple cold could have been fatal. At UCSF, Dr. Mort Cowan’s team collected Ja’Ceon’s … Continue reading Celebrating Ja’Ceon Golden
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