HIV used to deliver gene therapy cure

Scientists used a stripped down version of HIV to deliver a gene therapy payload into the DNA of two young patients suffering from a rare and fatal brain disease. The team says their approach will allow the modified gene to stay in the boys' stem cells, marking a cure that could have profound consequences for other people suffering from similar types of diseases.

"We think gene therapy could become a first-line treatment. It's definitely a strategy that could be applied to other conditions," said Nathalie Cartier, the study's lead author and research director at the French National Institute for Health and Medical Research in Paris.

Thalassemia and sickle cell anemia, as well as two neurological conditions, are next on the scientists' hit list. Their work with a modified version of HIV plays on the virus's extraordinarily ability to penetrate into the host's DNA and remain there, successfully eluding scientists for decades. After removing the toxic elements of HIV, it was used to correct the genetic defect that causes adrenoleukodystrophy, or ALD. After collecting blood from the patients' bone marrow a fully functional ABCD1 gene was inserted and then injected back into them.

- read the story from Bloomberg

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