In DepthBiomedicine

How safe is a popular gene therapy vector?

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Science  10 Jan 2020:
Vol. 367, Issue 6474, pp. 131
DOI: 10.1126/science.367.6474.131

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Summary

Just as gene therapy finally seems to be living up to its promise, a study has revived a lingering worry about the viral vessel that many efforts rely on to ferry therapeutic genes into patients. This "vector," a stripped-down version of adeno-associated virus (AAV), was thought to be safe because it rarely knits its cargo of human DNA into a cell's chromosomes, where it might activate cancer-causing genes. But a study of dogs with hemophilia, treated with AAV up to 10 years earlier, has shown that the vector can readily insert its payload into the host's DNA near genes that control cell growth. The new data suggest that by slipping into the chromosomes rather than floating free, the therapeutic DNA might have longer lasting benefits. But the findings are also fueling a long-running debate about whether AAV vectors could pose an unacceptable cancer risk.

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