Gene therapy has the potential to transform a terminal diagnosis – including my son’s
From our UK edition
It was nine years ago, in a windowless room in Great Ormond Street Hospital, that I first heard the words: ‘I think your son has something called Duchenne muscular dystrophy.’ I tried to comprehend what the consultant was telling me: the terminal diagnosis of my child. My son Eli, who looked like any other healthy three-year-old, had a broken gene inside him, the dystrophin gene. It meant that his muscles would stop working during his short lifetime. By 12 or 13 he could be in a wheelchair. In his late teens, in what should be the prime of his life, he would lose the use of his arms and hands. And sometime in his twenties, his heart and lungs would fail. His mind — his beautiful, inquisitive mind — was not affected.
