Disease Focus: Blood Disorders
Generation of long-term cultures of human hematopoietic multipotent progenitors from embryonic stem cells
For many therapeutic reasons it is important to have available large numbers of blood cells. However, it is difficult to generate large numbers of specialized blood cells that have the ability to neutralize autoimmunity and response to tumor cell growth. In this study we would develop a technique that would allow the production of large […]
Self-renewal and senescence in iPS cells derived from patients with a stem cell disease
The discovery of induced pluripotent stem (iPS) cell technology promises to revolutionize our understanding of human disease and to allow the development of new cellular therapies for regenerative medicine applications. The ability to reprogram a patient’s fibroblasts to iPS cells creates the opportunity to expand human cells with a specific genetic defect and to study […]
Stem Cell Gene Therapy for Sickle Cell Disease
Sickle cell disease (SCD), which results from an inherited mutation in the hemoglobin gene that causes red blood cells to “sickle” under conditions of low oxygen, occurs with a frequency of 1/500 African-Americans, and is also common in Hispanic-Americans, who comprise up to 5% of SCD patients in California. The median survival based on 1991 […]
Molecular Characterization and Functional Exploration of Hemogenic Endothelium
Hematopoietic cells are responsible for generating all cell types present in the blood and therefore critical for the provision of oxygen and nutrients to all the tissues in the body. Blood cells are also required for defense against microorganisms and even for the recognition and elimination of tumor cells. Because blood cells have a relatively […]
CURED: Stem Cell Clinical Trial Stories
Clinical trials are risky and rely on brave pioneers who are willing to put their bodies on the line to test something never before tried in people. At its December 2016 meeting, the governing Board of California’s Stem Cell Agency (CIRM) met four courageous heroes who have participated in CIRM-funded clinical trials and in two […]
Defeating Sickle Cell Disease with Stem Cells + Gene Therapy
Using a mix of science, humor and easy to understand language, this video describes sickle cell disease and a CIRM-funded clinical trial at UCLA that is testing a stem cell and gene therapy cure. This awful genetic disorder causes red blood cells to assume a sickle shape, clogging blood vessels and producing episodes of excruciating […]
Spotlight on Amyloidosis and Stem Cell Research: Robert Vescio MD – Cedars-Sinai
Dr. Robert Vescio spoke to CIRM’s governing board to promote awareness about amyloidosis, a rare, often fatal disease caused by the abnormal deposit of proteins called amyloids in various tissues of the body. Dr. Vescio, director of Cedars-Sinai’s multiple myeloma and amyloidosis program, explained the disease and described the current stem cell based treatment options. […]
Michael York: Amyloidosis and Stem Cell Research
Michael York, star of films such as Cabaret, Logan’s Run, and Austin Powers, spoke to the California stem cell agency governing board about his personal battle with amyloidosis, a rare, often fatal disease caused by the abnormal deposit of proteins called amyloids in various tissues of the body. In addition to amyloidosis, York was also […]