This report was first published by Endpoints News. To see the original version, click here
Five people with beta thalassemia who had their blood stem cells genetically altered no longer require regular blood transfusions to stay healthy. Their Chinese-made treatment is likely to be cheaper, and perhaps safer and more effective, than its American counterparts.
Developed by CorrectSequence Therapeutics, the therapy employs a novel form of CRISPR base editing designed to change a single letter of DNA with almost no unwanted and potentially dangerous off-target edits. The Shanghai-based company told Endpoints News that the first patient treated has now been transfusion-free for nearly two and a half years.
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