Feature

A second chance

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Science  03 Nov 2017:
Vol. 358, Issue 6363, pp. 582-585
DOI: 10.1126/science.358.6363.582

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Summary

The results of a gene therapy trial for a disease called spinal muscular atrophy that gradually paralyzes infants have blown away gene therapy researchers, marking one of the once-troubled field's most dramatic successes yet. All 15 babies treated, expected to die by age 2, are alive at 20 months or older, and most can sit up, according to a report this week. The news adds to the rising fortunes of gene therapy. But the study also breaks ground because it demonstrates the power of a virus carrying a therapeutic gene that, infused into a vein, can carry its genetic cargo straight to the central nervous system, across the so-called blood-brain barrier. The new treatment's apparent safety and success is emboldening other researchers to use gene therapy delivered into a vein or the spine to treat rare childhood neurological and muscular diseases, and even common adult disorders such as Parkinson's.