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The unprecedented study involves using the gene-editing technique CRISPR to edit a gene while it’s still inside a patient’s body. In exclusive interviews, NPR talks with two of the first participants.
Victoria Gray, 34, of Forest, Miss., has sickle cell disease. She is the first patient ever to be publicly identified as being involved in a study testing the use of CRISPR for a genetic disease.
This could be a crucial year for the powerful gene-editing technique CRISPR as researchers start testing it in patients to treat diseases such as cancer, blindness and sickle cell disease.
The insects were created, using CRISPR, to carry a powerful “gene drive.” The mosquitoes could provide a potent weapon against malaria, but they raise fears about unpredictable environmental effects.
Despite outrage over gene editing in China that affected the birth of twins, research is underway in the U.S. to assess the safety and effectiveness of CRISPR tools to edit genes in human embryos.