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In a milestone year, gene therapy finds a place in medicine
DNA manipulation finding success
W gene
In this Oct. 4 file photo, Dr. Albert Maguire, right, checks the eyes of Misa Kaabali, 8, at the Children's Hospital of Philadelphia. Misa was 4-years-old when he received his gene therapy treatment. On Tuesday, Dec. 19, 2017, the Food and Drug Administration approved the therapy which improves the vision of patients with a rare form of inherited blindness, another major advance for the burgeoning field of genetic medicine. - photo by Associated Press
After decades of hope and high promise, this was the year scientists really showed they could doctor DNA to successfully treat diseases. Gene therapies to treat cancer and even pull off the biblical-sounding feat of helping the blind to see were approved by U.S. regulators, establishing gene manipulation as a new mode of medicine.Almost 20 years ago, a teen's death in a gene experiment put a chill on what had been a field full of outsized expectations. Now, a series of jaw-dropping successes have renewed hopes that some one-time fixes of DNA, the chemical code that governs life, might turn out to be cures."I am totally willing to use the 'C' word," said the National Institutes of Health's director, Dr. Francis Collins.Gene therapy aims to treat the root cause of a problem by deleting, adding or altering DNA, rather than just treating symptoms that result from the genetic flaw.The advent of gene editing — a more precise and long-lasting way to do gene therapy — may expand the number and types of diseases that can be treated.
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