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Your search for “gene therapy” returned 715 results

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.

Friedmann Named 2015 Japan Prize Winner

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…

Southern California’s First Real-Time MRI-Guided Gene Therapy for Brain Cancer

August 6, 2013

Neurosurgeons at the University of California, San Diego School of Medicine and UC San Diego Moores Cancer Center are among the first in the world to utilize real-time magnetic resonance imaging (MRI) guidance for delivery of gene therapy as a potential treatment for brain tumors.

New Genetic ‘CopyCatchers’ Detect Efficient and Precise CRISPR Editing in a Living Organism

May 11, 2021

Scientists have developed a novel CRISPR-based genetic sensor called a “CopyCatcher” to detect instances in which a genetic element is copied precisely from one chromosome to another in cells of a fruit fly.

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…

How Loss of Single Gene Fuels Deadly Childhood Brain Cancer

September 10, 2020

UC San Diego researchers describe how the functional loss of a single gene negatively impacts neural development and promotes the growth of a particularly deadly form of pediatric brain cancer.

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.

First-in-Human Clinical Trial to Assess Gene Therapy for Alzheimer’s Disease

February 18, 2021

UC San Diego researchers have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimer’s disease or Mild Cognitive Impairment, a condition that often precedes full-blown dementia.

Enhancer RNAs Alter Gene Expression

June 3, 2013

…or “eRNAs” could provide a new way to alter gene expression in living cells, perhaps affecting the development or pathology of many diseases.

Engineers Develop Most Efficient Red light-activated Optogenetic Switch for Mammalian Cells

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…

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