Many CRISPR Gene Editing Clinical Trials Underway

Human clinical trials are using CRISPR/Cas9 to combat cancer and blood disorders. In these tests, researchers remove some of a person’s cells, edit the DNA and then inject the cells back in, now hopefully armed to fight disease. Researchers are also set to see how CRISPR/Cas9 works inside the human body. In an upcoming trial, people with an inherited blindness will have the molecular scissors injected into their eyes.

Future trials for Duchenne muscular dystrophy, cystic fibrosis and a wide variety of other genetic diseases will help millions of people worldwide.

University of Pennsylvania researchers have given two people with recurring cancers a CRISPR/Cas9 therapy. One person has multiple myeloma; the other, sarcoma. As part of an ongoing trial, both received T cells, a type of immune cell, programmed with CRISPR to stop cancer cells.

A clinical trial using CRISPR is treating an inherited type of blindness called Leber congenital amaurosis 10. A mutation in the CEP290 gene that leads to a nonfunctional protein. When the protein doesn’t work, rod cells in the retina die and light-gathering photoreceptors can’t renew themselves, resulting in blindness.

Editing as few as 10 percent of retinal cells may help restore some sight. In animal tests, CRISPR edited up to about 60 percent of cells in mice and almost 28 percent in monkeys, scientists reported in the February Nature Medicine.

In July, Editas and global pharmaceutical company Allergan opened recruitment for a blindness gene-editing trial. In the trial, two guide RNAs will lead Cas9 to make two cuts that will snip out the troublesome piece of DNA.