When No Treatment Exists, Parents Turn to Gene Therapy to Save Their Kids

Families of patients are starting advocacy groups, raising money for research, and founding biotech companies to advance cures for rare diseases.

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.

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