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Gene therapy treats children with rare diseases

Six kids are healthy, up to three years after treatment

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2:34pm, July 11, 2013

A virus derived from HIV can safely fix broken immune systems and correct genetic diseases, suggest two new studies involving children with rare conditions.

For both studies, researchers put healthy genes into the children’s own DNA using lentiviruses, in this case genetically engineered versions of HIV that can no longer cause disease. Earlier gene therapy trials using different viruses had a flaw: When the viruses plunked themselves into the patient’s DNA, they sometimes amped up activity of neighboring cancer-causing genes, leading to leukemia. That side effect, along with the death of a young man participating in another clinical trial, nearly halted gene therapy in the United States in the early 2000s.

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