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High-dose AAV gene therapy appears toxic in monkeys and piglets

Posted on: 6 February 2018, source: Science
A gene therapy trial that recently led to dramatic benefits for babies born with a fatal neuromuscular condition has raised hopes for using a similar approach to treat other diseases. But a new animal study suggests that the high doses (2x10E14 genome copies per kilogram body weight) of gene-carrying viruses used in such treatments may not always be as safe as the human clinical trial indicated. In the new research, the disclosure of which briefly sent the stock prices of several gene therapy companies plummeting yesterday, researchers injected a handful of young monkeys and pigs with many copies of adeno-associated virus 9 (AAV9), a normally harmless virus that infects neurons and is increasingly being used to ferry therapeutic genes into cells to treat neuromuscular diseases. Within days, some of the animals developed severe liver and neuron damage.