December 12, 2022
Using models, researchers at UC San Diego School of Medicine, with colleagues elsewhere, describe using RNA-targeting CRISPR/Cas13d technology to develop a new therapeutic strategy that specifically eliminates toxic RNA that causes Huntington’s Disease.
December 1, 2022
The California Institute for Regenerative Medicine has awarded UC San Diego researchers $4.8 million to advance a gene therapy to treat Friedreich’s ataxia, a rare but devastating neuromuscular disorder.
September 14, 2020
UC San Diego researchers demonstrate that one dose of their version of CRISR gene editing can chew up toxic RNA and almost completely reverse symptoms in a mouse model of myotonic dystrophy, a type of adult-onset muscular dystrophy.
December 23, 2020
UC San Diego and Ludwig Cancer Research scientists describe how a phenomenon known as “chromothripsis” breaks up chromosomes, which then reassemble in ways that ultimately promote cancer cell growth.
March 5, 2018
…researchers has developed a light-activated switch that can turn genes on and off in mammalian cells. This is the most efficient so-called “optogenetic switch” activated by red and far-red light that has been successfully designed and tested in animal cells—and it doesn’t require the addition of sensing molecules from outside…
August 15, 2017
Using fragments of circulating tumor DNA in blood, University of California San Diego School of Medicine researchers were able to identify theoretically targetable genetic alterations in 66 percent of patients with cancer of unknown primary (CUP), a rare disease with seven to 12 cases per 100,000 people each year.
January 28, 2015
Theodore Friedmann, MD, professor in the Department of Pediatrics at University of California, San Diego School of Medicine was named today one of three recipients of the 2015 Japan Prize, a prestigious international award honoring laureates whose “original and outstanding achievements in science and technology have advanced the frontiers of…
August 5, 2013
A team of scientists, led by Napoleone Ferrara, MD, has shown for the first time that a signaling protein involved in inflammation also promotes tumor resistance to anti-angiogenic therapy.
May 1, 2023
When alumni Michaelle Jinette and Kevin Witt’s fourth son Tristan was diagnosed with the rare KCNH1 genetic mutation, the couple raised money, collaborated with scientific partners, and initiated rare disease research on behalf of their child and the 50 others like him in the world.
April 1, 2024
UC San Diego researcher, Farah Sheikh, studies a genetic heart arrhythmia condition that usually strikes people under 40. Her research will be used in clinical trials of a treatment for ARV.
