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.