CRISPR infusion eliminates swelling in those with rare genetic disease

In a medical first, an infusion of the CRISPR gene editor into the blood of three people with a rare genetic disease is easing their symptoms, a biotech company reports. The experimental treatment tamped down a liver protein that causes painful and potentially life-threatening bouts of swelling in the throat and limbs. Two people in the company’s trial are doing so well after a single CRISPR injection that they no longer need drugs to control their condition. The data were reported at a meeting today in Berlin on the disease, called hereditary angioedema. The effort marks the second time the company, Intellia Therapeutics, has used in vivo delivery of CRISPR to inactivate a gene directly inside a person’s body. But the latest results reflect the first report of clinical benefits associated with injecting the tool, which can snip out or replace targeted bits of DNA, says John Leonard, Intellia president and CEO. The clinical data are "impressive" and "an important achievement for the field," says Fyodor Urnov, a CRISPR researcher at the University of California, Berkeley. CRISPR has already been shown to treat blood disorders via an ex vivo strategy in which a patient’s cells are harvested, edited in a lab, and then returned to the body . An in vivo approach for blindness disorders, where the gene editor is injected into the eye, is also showing tentative benefits . But directing CRISPR to specific organs or cells inside th...
Source: ScienceNOW - Category: Science Source Type: news