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The parents of a 2-year-old girl write that their daughter “could die within the next year” because a genetic mutation is causing her heart to fail.

“Time is quickly running out for me,” writes a man in his mid-30s whose DNA harbors a genetic mistake certain to destroy his brain within a matter of years.

“Watching my sons disintegrate before my eyes is heartbreaking,” writes a mother with two children affected by a faulty gene that affects cognition, speech and mobility. One of her sons, she writes, is still walking and in college, but “it is only a matter of time before he will be in a wheelchair and his cognition will decline.”

Stories of human tragedies like these arrive in my inbox with increasing, painful regularity. People write to see if I can build a medication to fix their genes and stave off an early, imminent death. Their wish is not futuristic: Many scientists, including me, build DNA fixes for a living.

Over the past decade, thousands of people have agreed to be genetically engineered in experimental trials to... see more