Gene Therapy
Gene-edited CAR-T cells show promise against aggressive T-cell leukemia
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Scientists at University College London and Great Ormond Street Hospital have developed a base-edited therapy called BE-CAR7 that uses universal CAR T-cells to treat relapsed or refractory T-cell acute lymphoblastic leukemia. Early trial results published in the New England Journal of Medicine and presented at the American Society of Hematology Annual Meeting indicate deep remissions in most patients, including those who did not respond to standard treatments, by tackling long-standing challenges in T-cell–based therapies.
A single injection of gene therapy has significantly improved hearing in all ten patients born with a genetic form of deafness, according to a new study. Researchers delivered a working copy of the OTOF gene into the inner ear, with most patients regaining hearing within one month. The treatment, tested in China, proved safe and effective across ages from one to 24.
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Researchers at the University of Waterloo have developed engineered bacteria designed to invade and eat solid tumors from the inside out. The approach uses microbes that thrive in oxygen-free environments, targeting the low-oxygen cores of tumors. A genetic modification allows the bacteria to survive near oxygenated edges, controlled by a quorum-sensing mechanism.