Photo Courtesy of Andrea Fernandez de Soto Imagine stepping outside and facing life‑threatening danger. For children born with ADA‑SCID (adenosine deaminase severe combined immunodeficiency), this is daily life. Their immune systems are so weak that a cold or a scraped knee can cause a dangerous infection. Donald Kohn, MD, a leading researcher at the UCLA … Continue reading Potential Gene Therapy for Children with ADA-SCID
SCID
How CIRM-funded research brings 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. Now, thanks to the work of researchers at UCLA Broad Stem Cell Research Center, a new lifesaving gene therapy could change all that. The five-year-old was born with ADA-SCID, a … Continue reading How CIRM-funded research brings lifesaving gene therapy to a 5-year-old
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
Bubble baby treatment cleared to restart clinical trial
Evie Vaccaro: Photo courtesy Nancy Ramos Three families battling a life-threatening immune disorder got some great news last week. A clinical trial that could save the life of their child has once again been given the go-ahead by the US Food and Drug Administration (FDA). Evie Vaccaro's Story The clinical trial is the work of … Continue reading Bubble baby treatment cleared to restart clinical trial
Clinical trial for SCID
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. SCID therapy … Continue reading Clinical trial for SCID
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, life is extremely challenging. SCID leaves them without a functioning immune system, so even minor infections can be life‑threatening. Without treatment, many children die within the first few years of life. CIRM-funded clinical trials CIRM‑funded stem cell and gene therapy trials at UCLA and UCSF/St. … 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 touted the potential of CRISPR, a gene-editing tool that lets you target a specific mutation and either cut it out or replace it with the corrected form of … Continue reading A better, faster, more effective way to edit genes
CIRM-Funded Study of Deadly Immune Disorder Gets Boost from FDA
Hataalii Begay, age 4, first child treated with UCSF gene therapy for Artemis-SCID When Hataalii Begay was born in a remote part of the Navajo nation he was diagnosed with a rare, usually fatal condition, Artemis-SCID. New stem cell therapy Today, thanks to a therapy developed at UCSF and funded by CIRM, he's a normal healthy … Continue reading CIRM-Funded Study of Deadly Immune Disorder Gets Boost from FDA
CIRM-funded stem cell clinical trial patients: Where are they now?
Ronnie with his parents Pawash Priyank and Upasana Thakur. Since its launch in 2004, the California Institute for Regenerative Medicine (CIRM) has been a leader in growing the stem cell and regenerative medicine field while keeping the needs of patients at the core of its mission. To date, CIRM has: Advanced stem cell research and therapy development for … Continue reading CIRM-funded stem cell clinical trial patients: Where are they now?
Good news on two CIRM-funded research on blood disorders
Dr. Rosa Bacchetta on the right with Brian Lookofsky (left) and Taylor Lookofsky after CIRM funded Dr. Bacchetta's work in October 2019. Taylor has IPEX syndrome It’s always lovely to end the week on a bright note and that’s certainly the case this week, thanks to some encouraging news about two CIRM-funded research on blood … Continue reading Good news on two CIRM-funded research on blood disorders