Gene therapy cures deadly childhood liver disease in mice, offering hope for ARC syndrome

ARC syndrome affects approximately six pregnancies annually in the UK, with affected children typically dying within their first year of life without treatment.

In a London laboratory, scientists have coaxed a dying liver back to life — at least in mice — offering the first credible hope for children born with ARC syndrome, a genetic disorder so severe that most do not survive their first year. The breakthrough, born from years of failure and refinement, centers on delivering a missing protein gene precisely to liver cells, sidestepping the cancer risk that had shadowed earlier attempts. It is a reminder that medicine's most consequential advances often arrive not in a single leap, but through the patient unlearning of what went wrong.

Gene therapy cures deadly childhood liver disease in mice, offering hope for ARC syndrome

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