Gene Therapy Trial Wrenches Families as One Child’s Death Saves Another
July 21, 2016
(MIT Technology Review) – The study, at the San Raffaele Telethon Institute for Gene Therapy, is proving dramatically effective in stopping metachromatic leukodystrophy, or MLD, an inherited disorder that strikes in childhood and destroys the brain’s white matter, leading to paralysis and dementia. The therapy involves adding a correct copy of a single gene to a child’s bone marrow. But it only works well if it’s given before symptoms develop: by the time most children, including Liviana, are diagnosed it’s too late. The exception is when a family is alerted by one sick child that others are at risk. Then genetic and biochemical tests can discover if there’s a disease threat in younger children.