Gene therapy trial wrenches families as one child’s death saves another

July 20, 2016

Alessandra Biffi

MIT Technology Review reports on a gene therapy trial in Italy that has reported strong early results for an inherited neurodegenerative disease that leads to paralysis, dementia and, ultimately, death. The trial led by Alessandra Biffi, MD – now director of gene therapy at Dana-Farber/Boston Children’s – helped a number of babies identified through newborn screening after an older sibling died of metachromatic leukodystrophy (MLD). The experimental gene therapy works best if treatment is given before symptoms appear. It appears to be the first gene therapy designed to be given preventively, Tech Review reports, and, according to recently published results, freezes or prevents the rapid onset of the disorder.


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