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Renewed Interest in Gene Therapy

Gene therapy has helped an increasing number of people with different genetic conditions, writes NPR's Shots blog.

It notes that the approach has been used to treat patients with spinal muscular atrophy and Leber's congenital amaurosis. "It's a very exciting time for the field," Carrie Wolinetz, associate director for science policy at the National Institutes of Health, tells NPR.

These recent successes have rekindled interest in gene therapy, NPR's Shots blog adds. Previous forays into gene therapy more than 10 years ago were beset by patients developing cancer and, in one case, dying, but Shots says that work researchers did to improve on viral delivery appears to be panning out. It adds that researchers are now exploring its use for a range of conditions.

Current gene therapy patients and their families seem "delighted" with it, Shots says. "I think that the gene therapy is a miracle," Christian Guardino, who was treated for Leber's congenital amaurosis, tells the blog. "I can't imagine what my life would be like without it."

Still, NPR's Shots blog notes that the treatments will be expensive. The first gene therapy approved by the FDA earlier this year to treat a form of leukemia has been priced at $475,000 and analysts estimate that others could cost $1 million.

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