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Researchers' persistence pays off as gene therapies yield results

Gene therapy has been tested in a small number of patients, and more longer-term studies are needed to judge safety and efficacy, but early results against devastating diseases are promising. Researchers have overcome setbacks and "have reached a point of maturation in the science and in some of the new approaches to gene therapy that have allowed us to make rapid advancements in a fairly short period of time," said NIH Associate Director for Science Policy Carrie Wolinetz.

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