What is gene therapy?

Gene therapy offers the opportunity to treat a wide range of chronic and severe inherited diseases that previously had few to no treatment options. Genetic diseases are caused by changes (called “mutations”) in the body’s genes. Gene therapy seeks to address the underlying genetic cause of an inherited disease by inserting a functional version of the mutated gene inside cells. Nicklaus Children’s is one of only a few facilities in Florida to offer ELEVIDYS, an FDA-approved treatment for ambulatory patients with Duchenne muscular dystrophy.

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Reviewed by: Migvis Monduy, MD

This page was last updated on: 1/29/2019 3:21:13 PM