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Your search for “gene therapy” returned 757 results

Finding Keys to Glioblastoma Therapeutic Resistance

August 25, 2014

…a deadly brain cancer – are resistant to drug therapy. The answer lies not in the DNA sequence of the tumor, but in its epigenetic signature. These findings have been published online as a priority report in the journal Oncotarget.

Personalized “Eye-in-a-Dish” Models Reveal Genetic Underpinnings of Macular Degeneration

May 9, 2019

Using stem cells derived from six people, UC San Diego School of Medicine researchers recapitulated retinal cells in the lab. This “eye-in-a-dish” model allowed them to identify genetic variants that cause age-related macular degeneration, a common cause of vision loss.

Genetic Adaptation Keeps Ethiopians Heart-Healthy Despite High Altitudes

August 3, 2015

…and hypoxia is the endothelin receptor type B (EDNRB) gene. Researchers at University of California, San Diego School of Medicine now find that mice with lower-than-normal levels of EDNRB protein are remarkably tolerant to hypoxia.

New Reprogramming Method Makes Better Stem Cells

July 2, 2014

…cells and could ultimately lead to improved stem cell therapies.

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.

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.

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.

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.

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.

Disjointed: Cell Differences May Explain Why Rheumatoid Arthritis Varies By Location

June 10, 2016

Researchers at the University of California San Diego School of Medicine, with colleagues in Pennsylvania and China, report that not only are there distinct differences in key cellular processes and molecular signatures between rheumatoid arthritis (RA) and osteoarthritis (OA) but, more surprisingly, there are joint-specific differences in RA. The findings…

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