Gene Therapy
Gene-edited CAR-T cells show promise against aggressive T-cell leukemia
Imeripotiwa na AI Picha iliyoundwa na AI Imethibitishwa ukweli
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.
An experimental gene therapy has demonstrated significant promise in slowing the progression of Huntington’s disease, a rare form of dementia, by about 75 percent in a late-stage trial. Researchers hailed the breakthrough as a major step forward, though challenges remain in delivery and regulatory approval. Efforts are underway to develop a more practical version of the treatment.
Imeripotiwa na AI Imethibitishwa ukweli
Scientists at Cedars-Sinai Medical Center have developed an experimental synthetic RNA drug called TY1 that strengthens the body’s ability to clear damaged DNA and promote tissue healing. Described in a paper in Science Translational Medicine, the treatment could improve recovery from heart attacks and certain inflammatory or autoimmune conditions by enhancing the activity of a key DNA-processing gene in immune cells.