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Alison Frase remembers the first moment when she thought gene therapy had a chance of curing patients like her son Joshua of a rare genetic muscle-wasting disease called X-linked myotubular myopathy. It was 2007, and she was viewing a video of a crippled mouse on her home computer. Four weeks earlier, the mouse had been injected with an engineered virus carrying a new strand of DNA intended to correct a genetic mutation that made its muscles limp and weak.

Frase watched in awe and began to cry as the mouse’s limbs started to twitch. Eventually, it picked itself up and walked for the first time. “I thought, who cries watching a video of a mouse?” she recalls.

Frase is very likely the reason why the same treatment is now about to be tested in humans. In recent years, gene therapies have become safer and better at hitting their intended targets in the body, leading to a handful of remarkable cures in clinical trials. Advocates for rare-disease patients—especially determined parents like Frase—are increasingly seeking to start gene-therapy programs. They are establishing patient advocacy organizations, raising money for research, and even founding their own biotechnology startups to find treatments where few or none currently exist.

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Image: via Flickr Attribution Some rights reserved by hottolengh

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