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
Stem cells
Gene Therapy Brings Hope to Families Affected by Rare Immune Disorder
Photo Courtesy of Andrea Fernandez de Soto Imagine a life where every time you step outside, everything in your environment poses a life-threatening risk. For children born with ADA-SCID (adenosine deaminase severe combined immunodeficiency), this is a reality. Their immune systems are so compromised that a common cold or a simple scrape on the knee … Continue reading Gene Therapy Brings Hope to Families Affected by Rare Immune Disorder
Two CIRM-funded research projects receive RMAT designation from FDA
The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to two research projects funded by the California Institute for Regenerative Medicine (CIRM). RMAT is a fast-track designation that can help speed up the development, review, and potential approval of treatments for serious or life-threatening diseases. The first RMAT designation … Continue reading Two CIRM-funded research projects receive RMAT designation from FDA
Stem Cell Awareness: What are stem cells and why research is important
Have you heard of stem cells but don't know what they are or why they are essential in medical research? In celebration of Stem Cell Awareness Day, the second Wednesday in October that is dedicated to educating about the importance of stem cells, the California Institute for Regenerative Medicine (CIRM) is providing this guide on … Continue reading Stem Cell Awareness: What are stem cells and why research is important
Transformative stem cell-based treatment for Cystic Fibrosis Sinusitis
A $6 million grant to Dr. Matthew Porteus and his team at Stanford University to develop an innovative stem cell-based gene correction therapy for chronic sinusitis in Cystic Fibrosis (CF) patients. CF is a common genetic disease that currently lacks a cure, causing patients to spend their lives managing symptoms. Despite significant advancements in CF … Continue reading Transformative stem cell-based treatment for Cystic Fibrosis Sinusitis
CIRM’s $31 million boost for cancer and eye disease research
The California Institute for Regenerative Medicine (CIRM) recently approved nearly $31 million in funding, further cementing our commitment to advancing the field of regenerative medicine. This substantial investment aims to drive clinical research targeting various cancers and eye diseases. CIRM’s dedication to fostering innovative treatments is demonstrated through its clinical program, which funds eligible stem … Continue reading CIRM’s $31 million boost for cancer and eye disease research
Investing in stem cell and gene therapy treatments for HIV
A recent article in Nature shared the news about a 53-year-old man in Germany who was declared free of HIV after receiving virus-resistant cells. The man—referred to as the “Düsseldorf patient”—was diagnosed with acute myeloid leukemia and underwent a stem cell transplant in 2013 that replaced his bone marrow cells with HIV-resistant stem cells from … Continue reading Investing in stem cell and gene therapy treatments for HIV
CIRM funds clinical trial to make cancer therapy safer, less toxic
Blood stem cell transplantation following high dose chemotherapy is standard of care and potentially curative for aggressive forms of lymphoma. However, this treatment regimen is limited by severe toxicity and life-threatening complications due to delayed recovery of the blood system and vascular related damage of multiple organs. Today the governing Board of the California Institute … Continue reading CIRM funds clinical trial to make cancer therapy safer, less toxic
CIRM-funding supports world’s first stem cell treatment for spina bifida delivered during fetal surgery
Dr. Diana Farmer (right) meets with Emily Lofton and her baby Robbie who had stem cell treatment for spina bifida in the womb. Photo: UC Davis Health The California Institute for Regenerative Medicine (CIRM) recently shared some encouraging news on The Stem Cellar about a CIRM-funded stem cell clinical trial for spina bifida at UC … Continue reading CIRM-funding supports world’s first stem cell treatment for spina bifida delivered during fetal surgery
CIRM-funded stem cell-gene therapy shows promise in ALS safety trial
Senior author of the study Clive Svendsen, PhD (center) With funding support from the California Institute for Regenerative Medicine (CIRM), Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. This combined stem cell and gene therapy can potentially protect diseased motor neurons … Continue reading CIRM-funded stem cell-gene therapy shows promise in ALS safety trial
The Stem Cellar 