In DepthBiomedicine

Gene therapy field hit by fresh safety concern

See allHide authors and affiliations

Science  09 Feb 2018:
Vol. 359, Issue 6376, pp. 621
DOI: 10.1126/science.359.6376.621

You are currently viewing the summary.

View Full Text

Log in to view the full text

Log in through your institution

Log in through your institution

Summary

A virus that buoyed the gene therapy field when it led to dramatic benefits in babies born with a fatal neuromuscular condition is under scrutiny. A small animal study suggests that high doses of the virus, called adeno-associated virus 9, can cause severe liver and neuron damage in young monkeys and pigs. The results drew attention in part because they come from the lab of James Wilson at the University of Pennsylvania, who led a 1999 trial in which a teenager died from an immune reaction to a different gene therapy vector. He and others say, however, that although researchers should watch for similar side effects, ongoing clinical trials with the gene carrier should not be halted.