Gene Therapy Trial Wrenches Families as One Child’s Death Saves Another

An important test of gene therapy in Italy is bringing joy and heartbreak to families afflicted with a rare brain disease by offering affected siblings unequal shots at life.

Amy Price, an American from Omaha, Nebraska, says that in 2011 she did something no mother should have to, leaving one sick toddler behind at a rented flat in Milan with caretakers while accompanying her younger infant for a life-saving treatment at an Italian hospital.  

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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.

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