Gene-Editing

A young Mississippi woman is thriving two years after getting treated for sickle cell disease with the revolutionary gene-editing technique known as CRISPR.

In a first, doctors injected the gene-editing tool CRISPR directly into cells in patients' eyes. The experiment helped these vision-impaired patients see shapes and colors again.

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.

Researchers edited the DNA in bone marrow cells taken from a Mississippi woman with sickle cell disease to produce a treatment that could alleviate the excruciating effects of her inherited illness.

NPR visited the only lab in the world known to be trying to use the powerful gene-editing tool CRISPR to modify the DNA in human sperm. If successful, it could be used to prevent genetic disorders.

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.