Gene therapy treats children with rare diseases | Science News

SUPPORT SCIENCE NEWS

Science News is a nonprofit.

Help us keep you informed.


News

Gene therapy treats children with rare diseases

Six kids are healthy, up to three years after treatment

By
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.

Now, researchers led by Luigi Naldini of the San Raffaele Telethon Institute for Gene Therapy in Milan have altered the lentiviruses so that they won’t accidently turn on nearby genes. The researchers then infect bone marrow stem cells with lent

This article is only available to Science News subscribers. Already a subscriber? Log in now. Or subscribe today for full access.

Get Science News headlines by e-mail.

More from this issue of Science News

From the Nature Index Paid Content