Gene therapy has been tested in a small number of patients, and more longer-term studies are needed to judge safety and efficacy, but early results against devastating diseases are promising. Researchers have overcome setbacks and "have reached a point of maturation in the science and in some of the new approaches to gene therapy that have allowed us to make rapid advancements in a fairly short period of time," said NIH Associate Director for Science Policy Carrie Wolinetz.
Researchers' persistence pays off as gene therapies yield results
Sign up for FasterCures SmartBrief
Medical research news
Get the intelligence you need: news and information that is changing your industry today, hand-curated by our professional editors from thousands of sources and delivered straight to your inbox.