Gene therapy gets a high-stakes test

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Science  15 Apr 2016:
Vol. 352, Issue 6283, pp. 286
DOI: 10.1126/science.352.6283.286

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A U.K. team is designing a clinical trial even more radical than prenatal stem cell therapy: the first ever test of gene therapy in pregnancy. The treatment aims to correct fetal growth restriction, in which blood flow to the placenta falters. Gene therapy has had a checkered history in children and adults, with side effects including cancer and at least one death. So rather than introduce new genes to the fetus itself, the team is targeting a blood flow–promoting gene to the uterine arteries.