November 10, 2021
Comparing features of a common laboratory fruit fly with its rarer cousin collected from Hawai’i Volcanoes National Park, UC San Diego researchers used CRISPR technology to uncover clues about how high-level control genes called Hox genes shape our appearance.
July 1, 2022
Scientists have developed a CRISPR-based technology that could offer a safer approach to correcting genetic defects. The new “soft” CRISPR system makes use of natural DNA repair machinery, providing a foundation for novel gene therapy strategies with the potential to cure genetic diseases.
May 9, 2019
Using stem cells derived from six people, UC San Diego School of Medicine researchers recapitulated retinal cells in the lab. This “eye-in-a-dish” model allowed them to identify genetic variants that cause age-related macular degeneration, a common cause of vision loss.
March 10, 2021
A gene therapy for chronic pain could offer a safer, non-addictive alternative to opioids. By temporarily repressing a gene involved in sensing pain, the treatment increased pain tolerance in mice, lowered their sensitivity to pain and provided months of pain relief without causing numbness.
March 20, 2017
Genetic mutations that cause cancer also weaken cancer cells, allowing researchers to develop drugs that will selectively kill them. This is called “synthetic lethality” because the drug is only lethal to mutated (synthetic) cells. Researchers at UC San Diego School of Medicine and Jacobs School of Engineering developed a method…
September 27, 2017
…mutations in the KRAS gene are among the most deadly. For decades, researchers have tried unsuccessfully to directly target mutant KRAS proteins as a means to treat tumors. Instead of targeting mutant KRAS itself, researchers at University of California San Diego School of Medicine are now looking for other genes…
September 27, 2016
…of transcription factors — regulatory proteins that activate some genes and inactivate others — drive the development of stem cells in the testes in mice. The investigators also linked RHOX gene mutations to male infertility in humans.
March 12, 2015
…California, San Diego School of Medicine have identified a gene variant that may be used to predict people most likely to respond to an investigational therapy under development for Alzheimer’s disease (AD). The study, published March 12 in Cell Stem Cell, is based on experiments with cultured neurons derived from…
April 2, 2018
The vast majority of genetic mutations associated with cancer occur in non-coding regions of the genome, yet it’s unclear how they may influence tumor development or growth. Researchers at University of California San Diego School of Medicine and Moores Cancer Center identified nearly 200 mutations in non-coding DNA that play…
May 4, 2021
Researchers at UC San Diego have used gene therapy to prevent learning and memory loss in a mouse model of Alzheimer’s disease, a key step toward eventually testing the approach in humans with the neurodegenerative disease.
