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A toddler born with a severe neuromuscular disease takes their first steps. A woman born blind sees a sunset emblazoned across a mountain valley her friends used to have to describe to her. A child born deaf hears their mother sing a soothing lullaby for the first time.
The advent of gene therapy has made these once science-fiction fantasies a near-reality. Instead of treating symptoms, gene therapy corrects the root cause of genetic disorders by inserting new genetic material into patients who have nonfunctional or harmful versions of…