August 27, 2013
…to stem cell and gene therapy which aim to advance the search for a cure. Cherqui is well-known in the cystinosis community, and serves on the Scientific Review Board of the Cystinosis Research Foundation. She was part of the scientific team that discovered the cystinosis gene and in 2009, her…
March 17, 2022
…also developed a CRISPR-based gene therapy for chronic pain, which could offer a safer and non-addictive alternative to opioids. The Mali lab has a strong translational focus with several gene therapy technologies licensed to and being further developed by startup companies including Navega Therapeutics and Shape Therapeutics, both co-founded by…
January 23, 2020
…efficacy of a unique gene therapy approach to treating cystinosis. The treatment was developed over more than a decade of research by Stephanie Cherqui, associate professor of pediatrics, and her team at University of California San Diego School of Medicine. “The day they started looking for people for the trial,…
February 10, 2022
…holds promise as a gene therapy for treating genetic diseases. In a proof of concept, UC San Diego researchers showed that the technology can treat a mouse model of Hurler syndrome, a rare genetic disease, by correcting its disease-causing mutation in RNA. The findings are published Feb. 10 in Nature…
June 26, 2020
…of stem cell and gene therapy for multi-systemic genetic disorders and the fundamental understanding of tissue repair by bone marrow stem cells. Stuart Lipton, adjunct professor of Molecular Medicine and professor of Neuroscience. Lipton’s work focuses on using genetically programmed human stem cells for transplantation as a potential cure for…
July 1, 2022
…a foundation for novel gene therapy strategies with the potential to cure a large spectrum of genetic diseases. In many cases, those suffering from genetic disorders carry distinct mutations in the two copies of genes inherited from their parents. This means that often, a mutation on one chromosome will have…
August 10, 2017
…are commonly used in gene therapy, but they are too small to hold Cas9 to target DNA. Yeo’s team made a smaller version of Cas9 by deleting regions of the protein that were necessary for DNA cleavage, but dispensable for binding RNA. “The main thing we don’t know yet is…
October 19, 2021
…Drug Administration has approved gene therapy for treating one form of IRD involving the gene RPE65, but for other IRDs caused by mutations in more than 280 different genes, there are no cures or treatments proven to slow disease progression. The researchers conducted whole-genome sequences (WGS) of 409 persons from…
March 28, 2013
…subjects, including stress reduction, gene therapy for heart repair, cancer prevention, successfully reducing menopause symptoms, and achieving longevity through diet and fitness. Keynote speaker Tahir Bhatti, MD, associate clinical professor in UC San Diego School of Medicine’s Department of Psychiatry, will share practical strategies on how to minimize stress in…
July 24, 2014
…the U.S. to perform magnetic resonance imaging–guided laser and gene therapy to treat brain cancers such as glioblastoma. To learn more about these approaches to brain surgery at UC San Diego Health System, click here.
