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For most of her life, Allison Corona lived in a world dimmed by bad genetic luck. A disease called Leber’s congenital amaurosis left her legally blind at age 4. She could not navigate the short distance from her driveway to her front door after dusk.

Three years ago, Corona, now 23, received an experimental medical treatment aimed at fixing the faulty genes in her eyes. Researchers at the Children’s Hospital of Philadelphia injected viruses carrying a good copy of her errant gene into her right eye and, nine days later, her left eye.

The world around her, once dark and austere, soon grew brighter. Her vision is still far from perfect, but for the first time, she sees that paper towels have texture. She marvels at the velvet floral wallpaper that covers her bedroom wall. She takes a college class that gets out at 10:30 p.m. and no longer fears getting stranded in the night.

“Things became much more beautiful for me,” Corona said.

First tested in patients a quarter-century ago, gene therapy — a risky approach aimed... see more