UC group launches clinical trial using CRISPR to correct sickle cell disease gene defect
April 1, 2021UCLA
Scientists at UCLA, UC San Francisco and UC Berkeley 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 patients’ own blood-forming stem cells.
The trial will combine CRISPR technology developed at the Innovative Genomics Institute a UC Berkeley–UCSF initiative founded by Berkeley’s Nobel Prize–winning scientist Jennifer Doudna with UCLA’s expertise in genetic analysis and cell manufacturing, and the decades-long expertise at UCSF Benioff Children’s Hospital Oakland in cord blood and marrow transplantation and in gene therapy for sickle cell disease. The four-year study will include six adults and three adolescents with severe sickle cell disease. It is planned to begin this summer in Oakland and Los Angeles.
April 1, 2021
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For thousands of years, people around the world have relied on medicinal folklore, herbal treatments and rituals to heal an array of ailments. Now, researchers from the University of California, Los Angeles (UCLA) have created an online platform featuring hundreds of thousands of these traditional therapies. Spanning seven continents and 200 years, the Archive of Healing draws on such sources as anthropologists’ field notes, scholarly journals, oral histories and folktales. (UCLA’s David Shorter was quoted.)
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March 12, 2021
Recent scientific advances have generated technologies that could harness the power of stem cell science to better understand and discover new treatments for neuropsychiatric disorders. On March 22, from 1 to 3 p.m., leaders from all areas of the field will discuss their research on human neuropsychiatric disorders.
Presented by the UCLA Broad Stem Cell Research Center, the UCLA Neuroscience Theme, the Depression Grand Challenge and the Semel Institute of Neuroscience and Human Behavior, this conference brings together leading scientists to discuss how human genetics and stem cell research can advance our understanding of the cellular and molecular mechanisms underlying neuropsychiatric disorders, and the implications of such research for reducing disparities in mental health.
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A City of Hope-led research team found that the same gene that increases the risk for Alzheimer s disease, ApoE4, can increase the susceptibility to and severity of COVID-19. Our study provides a causal link between the Alzheimer s disease risk factor ApoE4 and COVID-19 and explains why some (e.g., ApoE4 carriers) but not all COVID-19 patients exhibit neurological manifestations said Yanhong Shi, Ph.D., director of the Division of Stem Cell Biology at City of Hope and co-corresponding author of the new study. Understanding how risk factors for neurodegenerative diseases impact COVID-19 susceptibility and severity will help us to better cope with COVID-19 and its potential long-term effects in different patient populations.