A new gene therapy technique that uses high doses of viruses to carry healthy genes to cells recently brought relief to 15 babies suffering from a lethal neuromuscular condition. As the first human trial with high-doses of the AAV9 virus, its success gave a huge boost to the gene-therapy field.
“People are going to look back and see this as a milestone in a new type of medicine that’s going to have broad implications for lots and lots of diseases,” one researcher told Science last year.
However, those hopes were chilled this week when a leading gene-therapy researcher who was involved in the successful trial also published a study in which high-dose AAV9 infusions severely affected animals. Three young rhesus macaque monkeys developed liver failure and three piglets had motor neurone damage. Some of them had to be euthanised. The cause was not immediately apparent.
Continue reading: BioEdge: Enthusiasm for gene therapy cools after animal tests