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Your search for “gene therapy” returned 715 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.

Gene Editing Technique Helps Find Cancer’s Weak Spots

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…

Immune System Molecule Promotes Tumor Resistance to Anti-Angiogenic Therapy

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.

UC San Diego Biologists Discover Genes That Repair Nerves After Injury

September 21, 2011

…of California, San Diego have identified more than 70 genes that play a role in regenerating nerves after injury, providing biomedical researchers with a valuable set of genetic leads for use in developing therapies to repair spinal cord injuries and other common kinds of nerve damage such as stroke.

Friedmann Recognized for Pioneering Gene Therapy Research

January 29, 2015

Friedman Recognized for Pioneering Gene Therapy Research School of Medicine professor receives prestigious Japan Prize Theodore Friedmann 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…

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.

Putting ‘Super’ in Natural Killer Cells

June 11, 2020

Using induced pluripotent stem cells (iPSCs) and deleting a key gene, researchers at University of California San Diego School of Medicine have created natural killer cells — a type of immune cell — with measurably stronger activity against a form of leukemia, both in vivo and in vitro.

Researchers Find Fertility Genes Required for Sperm Stem Cells

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.

Gene Therapy Approach Shows Promise in Treating ALS

August 2, 2022

Researchers report that a gene therapy approach, developed at UC San Diego measurably delayed disease onset in humanized mouse and rat models of familial ALS, an inherited form of the disease that runs in families.

Gene Therapy Shows Promise in Treating Neuropathy from Spinal Cord Injuries

May 9, 2022

An international team of researchers, led by scientists at UC San Diego, report that a gene therapy that inhibits targeted nerve cell signaling effectively reduced neuropathic pain with no detectable side effects in mice with spinal cord or peripheral nerve injuries.

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