But scientists are still hopeful that they’re at the “cusp” of a breakthrough.
Scientists think they have achieved the first gene editing inside the body, altering DNA in adults to try to treat a disease, although it’s too soon to know if this will help. Preliminary results suggest that two men with a rare disorder now have a corrective gene at very low levels, which may not be enough to make the therapy a success. (2/7)
The first attempt at using genome editing to treat and cure patients with a rare, inherited disease has produced disappointing results in a small clinical trial. Despite the setback, Sangamo Therapeutics (SGMO), the biotech developing the genome-editing therapy, intends to keep trying. Transient improvements observed in a single patient suggest a more potent version of its treatment — already being readied for use — might be more effective, the company said. (Feuerstein, 2/7)
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