A 12 year-old Kurt Gillenberg. Photo courtesy of Kurt Gillenberg. When Kurt Gillenberg was 10 months old, his parents knew something wasn’t right. But it wasn’t until he reached 18 months that they found a doctor at the University of California, San Diego, who diagnosed Kurt with cystinosis – a rare genetic, metabolic defect that … Continue reading Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment
Rare Diseases
Bringing a community together at the 2nd Annual ALSP Conference
In addition to funding innovative programs and research that accelerate the development of stem cell and gene therapy treatments, the California Institute for Regenerative Medicine (CIRM) provides funding to support valuable mission-specific scientific conferences where people can meet to exchange scientific information to meet goals relevant to CIRM’s mission. Recently, CIRM supported the 2nd Annual … Continue reading Bringing a community together at the 2nd Annual ALSP Conference
How CIRM-funded research is bringing a lifesaving gene therapy to a 5-year-old
For her first year of life, Seersha Sulack stayed mostly in her bedroom because something as simple as a common cold could have killed her. The five-year-old was born with ADA-SCID, a condition so rare that only eight babies a year are born with it in the United States. Now, thanks to the work of … Continue reading How CIRM-funded research is bringing a lifesaving gene therapy to a 5-year-old
Join the movement to fight rare diseases
Tomorrow, February 28th, is Rare Disease Day. It’s a day to remind ourselves of the millions of people, and their families, struggling with these diseases. These conditions are also called orphan diseases because, in many cases, drug companies were not interested in adopting them to develop treatments. Here at the California Institute for Regenerative Medicine (CIRM), we … Continue reading Join the movement to fight rare diseases
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
Making transplants easier for kids, and charting a new approach to fighting solid tumors.
Every year California performs around 100 kidney transplants in children but, on average, around 50 of these patients will have their body reject the transplant. These children then have to undergo regular dialysis while waiting for a new organ. Even the successful transplants require a lifetime of immunosuppression medications. These medications can prevent rejection but … Continue reading Making transplants easier for kids, and charting a new approach to fighting solid tumors.
Sweet 16 and counting for stem cell clinical trial
Dr. Judy Shizuru: Photo courtesy Jasper Therapeutics Over the years the California Institute for Regenerative Medicine (CIRM) has invested a lot in helping children born with severe combined immunodeficiency (SCID), a fatal immune disorder. And we have seen great results with some researchers reporting a 95 percent success rate in curing these children. Now there’s … Continue reading Sweet 16 and counting for stem cell clinical trial
Fast Track Designation for a therapy making transplants safer for children with a fatal immune disorder
Bone marrow transplant For children born with severe combined immunodeficiency (SCID) life can be very challenging. SCID means they have no functioning immune system, so even a simple infection can prove life threatening. Left untreated, children with SCID often die in the first few years of life. There are stem cell/gene therapies funded by the … Continue reading Fast Track Designation for a therapy making transplants safer for children with a fatal immune disorder
A better, faster, more effective way to edit genes
Clinical fellow Brian Shy talks with postdoctoral scholar Tori Yamamoto in the Marson Lab at Gladstone Institutes on June 8th, 2022. Photo courtesy Gladstone Institutes. For years scientists have been touting the potential of CRISPR, a gene editing tool that allows you to target a specific mutation and either cut it out or replace it … Continue reading A better, faster, more effective way to edit genes
Stem Cell Agency Expands Industry Alliance Program to Accelerate Therapies
An ever-growing array of academic and industry resources are required to rapidly translate scientific discoveries and emerging technologies toward safe and effective regenerative medicine therapies for patients. To help, the California Institute for Regenerative Medicine (CIRM) is creating a network of Industry Resource Partners (IRP) that will make its unique resources available to help accelerate … Continue reading Stem Cell Agency Expands Industry Alliance Program to Accelerate Therapies