Gene therapy setback? Animal deaths in treating muscular dystrophy spark new concerns over high doses

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.

1-31-2018 dr-james-wilson_1024xx2400-1354-0-296That’s where the danger could come in, says Wilson. At those doses, he says, his team found that two different viruses caused extreme and sudden immunological effects.


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.

Read full, original post: The doctor responsible for gene therapy’s greatest setback is sounding a new alarm

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