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Your search for “gene therapy” returned 741 results

Study Provides Hope for Some Human Stem Cell Therapies

August 20, 2015

An international team of scientists headed by biologists at UC San Diego has discovered that an important class of stem cells known as human “induced pluripotent stem cells,” or iPSCs, which are derived from an individual’s own cells, can be differentiated into various types of functional cells with different fates…

New Version of DNA Editing System Corrects Underlying Defects in RNA-based Diseases

August 10, 2017

Until recently, CRISPR-Cas9 gene editing could only be used to manipulate DNA. In 2016, University of California San Diego School of Medicine researchers repurposed the technique to track RNA in live cells in a method called RNA-targeting Cas9. In a study published August 10 in Cell, the team took RCas9…

Gene Therapy Reverses Effects of Autism-Linked Mutation in Brain Organoids

May 2, 2022

UC San Diego scientists use lab-grown human brain tissue to identify neural abnormalities in Pitt-Hopkins Syndrome and show gene therapy tools can rescue neural structure and function.

Boosting A Natural Protection Against Alzheimer’s Disease

March 12, 2015

…California, San Diego School of Medicine have identified a gene variant that may be used to predict people most likely to respond to an investigational therapy under development for Alzheimer’s disease (AD). The study, published March 12 in Cell Stem Cell, is based on experiments with cultured neurons derived from…

New Biomarker Predicts Whether Neurons Will Regenerate

October 16, 2023

Researchers from University of California San Diego have identified a new biomarker that can predict whether or not neurons will regenerate after an injury. The findings could help scientists develop regenerative therapies for spinal cord injuries and other neurological conditions.

HIV Infection Prematurely Ages People by an Average of Five Years

April 21, 2016

Thanks to combination antiretroviral therapies, many people with HIV can expect to live decades after being infected. Yet doctors have observed these patients often show signs of premature aging. Researchers at University of California San Diego School of Medicine and the University of Nebraska Medical Center have applied a highly…

Non-Coding DNA Located Outside Chromosomes May Help Drive Glioblastoma

November 21, 2019

According to a new Cell study, extra DNA scooped up and copied alongside cancer-causing genes helps keep tumors going — elements that could represent new drug targets for brain tumors and other cancers notoriously difficult to treat.

UC San Diego Chemists Use Light to Pinpoint Gene Expression

February 13, 2018

…graduate students activated cellular gene expression with unique precision. By modifying messenger RNA (mRNA)—a group of molecules that carries genetic information from DNA to ribosomes where specifications of gene expression occur—the chemists were able to precisely trigger gene expressions at a specific time and place using laser light. This novel…

A New Approach to Faster Anticancer Drug Discovery

March 13, 2012

Tracking the genetic pathway of a disease offers a powerful, new approach to drug discovery, according to scientists at the University of California, San Diego School of Medicine who used the approach to uncover a potential treatment for prostate cancer, using a drug currently marketed for congestive heart failure.

Researchers Expand Clinical Trials Investigating Convalescent Plasma Therapy for COVID-19

January 28, 2021

…Trials Investigating Convalescent Plasma Therapy for COVID-19 It’s hoped the long-used approach, which would use antibody-rich infusions, can either reduce disease symptoms or prevent infections after exposure to SARS-CoV-2 Parallel to COVID-19 vaccination efforts, physicians and researchers at UC San Diego Health, with collaborators across the country, are conducting a…

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