Tag: editing

Using base editing to treat sickle cell disease in mice

A team of researchers from the Broad Institute, Harvard University and St. Jude Children’s Research Hospital has used a base editing technique in a novel way to treat sickle cell disease (SCD) in mice. In their paper published in the journal Nature, the group describes their new technique and how well it worked when tested

RNA editing protein ADAR1 protects telomeres and supports proliferation in cancer cells

Scientists at The Wistar Institute identified a new function of ADAR1, a protein responsible for RNA editing, discovering that the ADAR1p110 isoform regulates genome stability at chromosome ends and is required for continued proliferation of cancer cells. These findings, reported in Nature Communications, reveal an additional oncogenic function of ADAR1 and reaffirm its potential as

Editing RNA delivers precision strike on triple-negative breast cancer

The move toward targeted anti-cancer treatments has produced better outcomes with fewer side-effects for many breast cancer patients. But so far, advances in precision medicine haven’t reached people diagnosed with so-called triple-negative breast cancer. An innovative compound developed in the lab of Scripps Research chemist Matthew D. Disney, Ph.D., offers a new potential route to

Gene editing for recessive dystrophic epidermolysis bullosa: A little bit closer to clinical applications

A group of researchers from the Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) (Biomedical Research Networking Centre on Rare Diseases), Universidad Carlos III de Madrid (UC3M), the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) (Research Center for Energy, Environmental and Technology), and the Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD)

With gene editing, researchers cure blood disorder in fetal mice

With the combined efforts of three Yale laboratories, researchers conducted the first demonstration of site-specific gene editing in a fetus, correcting a mutation that causes a severe form of anemia. The technique, described in a paper published June 26 in Nature Communications, involves an intravenous injection of nanoparticles carrying a combination of donor DNA and