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Your search for “gene therapy” returned 728 results

Even DNA that Doesn’t Encode Genes Can Drive Cancer

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…

New Gene Fusions and Mutations Linked to Gastrointestinal Stromal Tumors

December 15, 2016

…researchers have identified specific gene mutations linked to gastrointestinal stromal tumors (GIST), which primarily occur in the stomach or small intestine, but 10 to 15 percent of adult GIST cases and most pediatric cases lack the tell-tale mutations, making identification and treatment difficult. Researchers at University of California San Diego…

Editing Genes One by One Throughout Colorectal Cancer Cell Genome Uncovers New Drug Targets

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…

New Biomarker Identified in Women with Mental Illness

June 19, 2015

…report identifying a biological marker: the over-production of specific genes that could be a diagnostic indicator of mental illness in female psychiatric patients.

Gene Identified That May Provide Potential Therapy for Cerebral Cavernous Malformations

October 9, 2017

Researchers at University of California San Diego School of Medicine, with national collaborators, have identified a series of molecular clues to understanding the formation of cerebral cavernous malformations (CCMs). The study offers the first genome-wide analysis of the transcriptome of brain microvascular endothelial cells after KRIT1 inactivation.

Tethering of Shattered Chromosomal Fragments Paves Way for New Cancer Therapies

June 15, 2023

UC San Diego scientists discover shattered chromosomal fragments are tethered together during cell division before being rearranged; destroying the tether may help prevent cancerous mutations

Next Steps in Potential Stem Cell Therapy for Diabetes

January 10, 2013

…with the longer-term goal of developing new stem cell therapies for diabetes.

CRISPR Technology Improves Huntington’s Disease Symptoms in Models

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.

Scientists Receive $4.8M to Pursue Gene Therapy for ‘Incurable’ 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.

Twist on CRISPR Gene Editing Treats Adult-Onset Muscular Dystrophy in Mice

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.

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