NPR

Gene Therapy Shows Promise For A Growing List Of Diseases

After decades of hope and disappointment, doctors have now been able to treat several different types of genetic conditions by giving each patient a healthy version of their defective gene.
Source: Pasieka

Eli Wheatley and Christian Guardino are among a growing number of patients whose lives are apparently being saved or radically improved by gene therapy.

Wheatley, 3, of Lebanon, Ky., and Guardino, 17, of Patchogue, N.Y., were both diagnosed with what were long thought to be incurable genetic disorders. In the past, Wheatley's condition would have probably killed him before his first birthday. Guardino's would have blinded him early in life.

But after receiving experimental gene therapies, both seem to be doing fine.

"It's a very exciting time for the field," says , the associate director for science policy

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