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UCSF is launching a new initiative to propel the development of living therapeutics – a category of treatments broadly defined as human and microbial living cells that are selected, modified, or engineered to treat or cure disease – and bring them quickly to patients.
Scientists at UC San Francisco, UC Berkeley and UCLA have received U.S. Food and Drug Administration approval to jointly launch an early phase, first-in-human clinical trial of a CRISPR gene correction therapy in patients with sickle cell disease using the patient’s own blood-forming stem cells.
UCSF scientists have discovered a new way to control the immune system’s “natural killer” cells, a finding with implications for novel cell therapies and tissue implants that can evade immune rejection.
Giant lizards with superpowered hearts. Hairless rodents that don’t seem to age. Songbirds that babble like human babies. These and other scurrying, soaring, and slithering wonders are teaching scientists how our own bodies work – and how to fix them.
Administering stem cell or enzyme therapy in utero may be a path to alleviating some congenital diseases that often result in losing a pregnancy, according to a new study in mice by UCSF researchers. They showed that stem cells can enter the fetal brain during prenatal development and make up for cells that fail to make an essential protein.
Advances in medicine and public health have dramatically extended the lifespan of hearts, lungs, and other vital organs. But for women, the ovaries remain a stubborn exception. That may soon change, says fertility expert Marcelle Cedars.
International team of researchers report progress in using stem cells to develop new therapies for Pelizaeus-Merzbacher disease, a rare genetic condition affecting boys that can be fatal before 10 years of age.
In a paper researchers describe a technique that uses a special version of CRISPR developed at UCSF to systematically alter the activity of genes in human neurons generated from stem cells, the first successful merger of stem cell-derived cell types and CRISPR screening technologies.