Matthew Bishop was told there was no treatment that could save his vision. But now scientific breakthroughs in gene therapy have given him, and others, hope
In his office in Oxford’s John Radcliffe hospital, Prof Robert MacLaren sits upright, his back as straight as a soldier’s, and tells me about the lowest point in his 20-year career. It was the rejection, many years ago, of his grant application for a project investigating how gene therapy might treat conditions causing blindness. “It was completely panned by the reviewers,” he says. “We were told ‘There’s no way it’s ever going to happen – it’s a complete waste of time funding such a ridiculously stupid project’.”
In October last year, MacLaren successfully completed the world’s first gene therapy trial for one such condition, called choroideremia, as part of the largest late-stage trial ever for any genetic disease. It marks an extraordinary breakthrough in the quest of scientists and clinicians to understand why and how our own genes can make us ill, and the apparently miraculous possibility of rewriting our genetic code. But MacLaren is understated about this victory: “It’s really satisfying, when you’re given such a rebuttal, to then prove the reviewers wrong. I’d love to go back to them and say: Look what’s going on now.”
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