Gene Therapy

About 350 million people suffer from rare diseases, most of which are genetic. But the individual disorders affect relatively few people, so there’s little commercial incentive to bring these treatments to market.

Central Florida has one of the highest yearly diagnosis averages of Sickle cell disease in the country, but a recently approved gene-editing treatment might bring relief.

The teen was born with a rare genetic condition that causes blisters all over his body and in his eyes. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. His doctor wondered if it could be adapted for his eyes.

Researchers at the University of Florida have developed a new therapy that they hope could lead to a cure for multiple sclerosis.The process uses a gene…