An influential scientist involved in gene therapy’s biggest setback, the death of a study volunteer 19 years ago, has issued a surprise warning over the dangers of the gene-replacement technique.
James Wilson of the University of Pennsylvania reported ... that monkeys and pigs given super-high doses of gene therapy died.
The warning comes amidst a scramble by three companies—Sarepta Therapeutics, Pfizer, and Solid Biosciences—to be the first to use the technique to cure muscular dystrophy. That disease strikes young boys, destroys their muscles, and kills them by their 20s.
To attack the disease, researchers replace patients’ damaged copies of a gene called dystrophin by introducing viral particles that carry a correct copy. Reaching the countless muscle cells in a boy’s body requires extremely high doses of these particles—400 trillion or more per pound of body weight.
Studies of the extra-high doses have only recently begun. Among them, Wilson said in his paper, are the one initiated by Solid Biosciences as well as a study at Nationwide Children’s Hospital, in Columbus, Ohio, where at least 15 infants got megadoses in a successful effort to treat a different disorder, spinal muscular atrophy.
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