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Your search for “Gene Therapy” returned 762 results

New Resource Makes Gene Editing Technology Even More User-Friendly

July 17, 2015

…to accompany the powerful gene editing tool called CRISPR/Cas9, which has been widely adopted to make precise, targeted changes in DNA. This breakthrough has the potential to facilitate new discoveries in gene therapies and basic genetics research. The research was published in the July 13 issue of Nature Methods.

How Gene Therapy May Hold Key to Treating Life-Threatening Cardiac Disease

March 18, 2020

New study finds gene therapy improved cardiac, muscle and liver function in Danon disease mouse models.

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.

Molecular Tumor Board Helps in Advanced Cancer Cases

May 5, 2014

With accelerating development of personalized cancer treatments matched to a patient’s DNA sequencing, proponents say frontline physicians increasingly need help to maneuver through the complex genomic landscape to find the most effective, individualized therapy.

Gene Variant Identified for Kawasaki Disease Susceptibility

February 9, 2017

…of Medicine, with colleagues at Rady Children’s Institute for Genomic Medicine and in London and Singapore, have conducted novel whole genome sequencing of a family in which two of four children were affected by Kawasaki disease. They have identified plausible gene variants that predispose some children to developing the disease.

Genomic Study Revealing Among Diverse Populations with Inherited Retinal Disease

October 19, 2021

An international team of researchers has broadened and deepened understanding of how inherited retinal dystrophies (IRDs) affect different populations of people and, in the process, have identified new gene variants that may cause the diseases.

“Wildly Heterogeneous Genes”

September 16, 2013

…share the exact same genetic mutations, a fact that has confounded scientific efforts to better categorize cancer types and develop more targeted, effective treatments. Researchers at the University of California, San Diego propose a new approach called network-based stratification, which identifies cancer subtypes not by the singular mutations of individual…

Researchers ID Cancer Gene-Drug Combinations Ripe for Precision Medicine

July 21, 2016

…the number of cancer gene mutations that can be specifically targeted with personalized therapies, researchers at University of California San Diego School of Medicine and Moores Cancer Center looked for combinations of mutated genes and drugs that together kill cancer cells. The study, published July 21 in Molecular Cell, uncovered…

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…

Altered Cell Cycle Gene Activity Underlies Brain Overgrowth in Autistic Toddlers

December 14, 2015

Further underscoring the prenatal origins of Autism Spectrum Disorder (ASD), researchers at University of California, San Diego School of Medicine describe for the first time how abnormal gene activity in cell cycle networks that are known to control brain cell production may underlie abnormal early brain growth in the disorder.

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