Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.
Researchers have created a novel gene therapy approach that shows promise for someday treating an eye disease that results in progressive vision loss.
Scientists have used metal-organic frameworks to effectively deliver the CRISPR/Cas9 genetic snipping tool into human cancer cells.
According to researchers from McMaster University and the Montreal Clinical Research Institute, they have identified a “fountain of youth” in a rare genetic marker that is unique to a few French-Canadian families.
Researchers affiliated with the Center for Cell-Based Therapy (CTC) in Ribeirão Preto, Brazil, have identified for the first time a non-hereditary mutation in blood cells from a patient with GATA2 deficiency, a rare autosomal disease caused by inherited mutations in the gene that encodes GATA-binding protein 2 (GATA2).
Light-activated liposomes could help to deliver CRISPR gene therapy - and the method could prove safer and more direct than current methods.
Scientists have deigned a new method to inhibit a toxicity observed in the sensory neurons of DRG following gene therapy to treat neurological diseases.
The thyroid hormone used to promote the proliferation of hepatocytes improved the efficiency of CRISPR/Cas9-mediated gene repair in the mouse liver.
Five research projects with exceptional promise to deliver new life-changing and health-altering therapies have received the inaugural Blavatnik Therapeutics Challenge Awards (BTCA) at Harvard Medical School.
Gene editing for the development of new treatments, and for studying disease as well as normal function in humans and other organisms, may advance more quickly with a new tool for cutting larger pieces of DNA out of a cell's genome, according to a new study by UC San Francisco scientists.
Natural killer T (NKT) cells, a type of immune cells known for their potent anti-cancer properties in murine tumor models, have been developed into a novel form of immunotherapy to treat patients with cancer.
Gene therapy offers an excellent potential for treating specific types of genetic defects and cancer, immunological diseases, infections, and wounds.
There is great potential in gene therapy for treating certain types of cancer and genetic defects, immunological diseases, wounds and infections.
Until now, seven genes were known to be involved in hearing loss in Israel's Jewish population. A new study led by Zippora Brownstein, PhD, and Prof. Karen Avraham from the Sackler Faculty of Medicine at Tel Aviv University has found that 32 genes are responsible for inherited hearing loss in Israeli Jewish families.
Gene therapy was successfully used to overcome the cardiac effects of Freidreich’s ataxia (FA) in a mouse model of the disease, as reported in the peer-reviewed journal Human Gene Therapy. Click here to read the full-text article free online through October 18, 2020.
Scientists made a comparison of their developed carriers for delivery of genome editing (GE) tools with other available analogs.
Introducing CRISPR in a Box™ - it's not a new crunchy cereal or the latest snack - it's an educational tool kit consisting of state-of-the-science gene editing technology that could be coming to a school near you.
Retinitis pigmentosa is the most prevalent form of congenital blindness. Using a retinitis pigmentosa mouse model, researchers from Ludwig-Maximilians Universitaet (LMU) in Munich have now shown that targeted activation of genes of similar function can compensate for the primary defect.
Postnova Analytics has published an applications report that presents data from a collaboration with the Analytical Development group at Biogen Inc. on separation of a virus mixture using Asymmetrical Flow Field-Flow Fractionation and measurement of their radius of gyration.
In May 2019, the U.S. Food and Drug Administration (FDA) approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy (SMA).
Personalized cancer treatments are no longer just options of the future. In the past few years, researchers have made significant progress in 'teaching' the body's immune T cells to recognize and kill specific cancer cells, and human clinical trials have shown that this approach can successfully eliminate tumors