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Gene Therapy Death May Delay New Trials

Researchers hoping to test a novel nonviral gene therapy system in people next year are unsure of approval prospects, following the recent death of a young man who was treated with an experimental viral vector.1 The man's health deteriorated rapidly a day after he was treated with an adenovirus carrying a therapeutic gene to treat ornithine transcarbamylase (OTC) deficiency. R. Michael Blaese Instead of taking the conventional gene therapy approach of delivering missing genes, the novel approa

Paul Smaglik

Researchers hoping to test a novel nonviral gene therapy system in people next year are unsure of approval prospects, following the recent death of a young man who was treated with an experimental viral vector.1 The man's health deteriorated rapidly a day after he was treated with an adenovirus carrying a therapeutic gene to treat ornithine transcarbamylase (OTC) deficiency.


R. Michael Blaese
Instead of taking the conventional gene therapy approach of delivering missing genes, the novel approach would use chimeraplasts--oligonucleotides containing both DNA and RNA that theoretically correct point mutations by initiating the cell's DNA mismatch repair machinery2--to treat Crigler-Najjar (C-N) syndrome. Children with C-N lack the complete protein necessary to break down toxic bilirubin in the liver and must spend 12-18 hours a day under a blue light to clear bilirubin. Even with that light therapy, their prospects to live into adulthood without a liver transplant...

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