CRISPR Gene-Editing Tool Used Inside Humans for the First Time

Arthur T Knackerbracket has found the following story:

In a world first, CRISPR, the powerful gene-editing tool that can cut and paste DNA, has been used inside the human body for the first time. Scientists at the Casey Eye Institute at Oregon Health & Science University, Portland, have administered a new CRISPR-based medicine to treat an inherited form of blindness, according to the two biotech companies which make the treatment.

"This dosing is a truly historic event -- for science, for medicine, and most importantly for people living with this eye disease," said Cynthia Collins, president and CEO of Editas Medicine, a gene-editing company headquartered in Massachusetts.

The first patient in the trial received a dose of the experimental drug, called AGN-151587, via an injection in the eye. The idea is that it delivers the gene-editing tool CRISPR directly to cells in the eye which are affected by the genetic disease. CRISPR is able to find its way into those cells and correct the gene -- a cut-and-paste scenario that sees a tiny DNA edit made to remove the mutation.

Importantly, the CRISPR edit is permanent, which means patients may only need a single dose and be set for life.

The trial is expected to enroll 18 patients in total and will look at different doses of the experimental drug, refining how much is necessary to achieve the goal of reversing blindness -- without any side effects. Information about the first patient is scant, with researchers staying silent on patient information and when the surgery officially occurred.

From the press release:

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