September 8, 2016
(STAT News) – Researchers have cleared the last scientific hurdle to a clinical trial of gene therapy to cure sickle cell disease, they reported on Tuesday, fueling hopes that they will begin enrolling patients early next year. But they dodged a bullet. The new study, conducted in mice, addressed a sometimes calamitous risk in gene therapy: the difficulty of changing only one thing when tweaking the DNA of a cell.
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