Scientists first proposed treating disease by changing the DNA in a person’s cells nearly 50 years ago. Today, the promise of gene therapy is beginning to be realized. Currently, there are nearly 20 gene therapy medicines approved by the FDA and over a thousand registered clinical trials involving gene therapy. With robust and predictable NIH funding, these numbers will continue to grow.

Read more in my fact sheet for the American Society of Human Genetics: Gene Therapy: From Fundamental Research to Clinical Success.