The doctor responsible for gene therapy’s greatest setback is sounding a new alarm - MIT Technology Review
►https://www.technologyreview.com/s/610141/the-doctor-responsible-for-gene-therapys-greatest-setback-is-sound
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.
That’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, including damage to the liver and blood vessels.
[...] The general type of #virus implicated, called an AAV, is widely used and considered safe. More than 2,000 people have been treated with AAVs, which have been used in successful tests of gene-therapy treatments for hemophilia and blindness.
Thanks to those successes, gene therapy has been racing forward, building toward ever more dramatic cures. The latest tests move into unknown territory because they involve much higher doses (which are needed to reach muscles or brain cells) and because the subjects are children.