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As the first patient to receive an experimental treatment that relied on the gene-editing technique CRISPR continues to do well 17 months later, more patients seem to be benefiting, too.
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Doctors used CRISPR to edit genes of cells inside a patient's eye, hoping to restore vision to a person blinded by a rare genetic disorder. A similar strategy might work for some brain diseases.
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NPR tells the exclusive, behind-the-scenes story of the first person with a genetic disorder to be treated in the United States with the revolutionary gene-editing technique CRISPR.
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She's the first patient with a genetic disorder to be treated with the powerful gene-editing technique CRISPR. The treatment has wrapped up, and now she's waiting to see if it brings relief.