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gene therapy

Akintunde Odunsi is used to taking long, brisk walks. So he worried in 2021 when he started feeling so tired and short of breath that he had to turn back after a few minutes.  The retired stockbroker, 73 years old, chalked up the fatigue to grief over the death of his sister. His cardiologist found a different cause: a genetic mutation causing clumps of malformed proteins to build up in his heart, taxing its ability to pump blood. 

Without treatment, he would die. After a desperate search, his family found a promising but risky option: an experimental drug that would edit his DNA inside his body to stop his disease, which is called transthyretin amyloidosis.

Odunsi decided it offered his best chance. “Go for it,” he told himself. 

It sounds like science fiction, but Odunsi is among dozens of people participating in studies on a controversial new forefront of the gene-editing revolution. 

Regulators last year approved the world’s first medicine using Crispr, the Nobel Prize-winning tool for modifying genes. The medicine, for sickle-cell disease, a group of inherited blood...