HIV virus and gene therapy

Gene therapy had a success this week due to the HIV virus. Two young boys have been treated for ALD, a brain disease, with a modified HIV virus.


From the New York Times article, After Setbacks, Small Successes for Gene Therapy:

The new approach involved using HIV, the virus that causes AIDS, to insert genes, modifying the virus first so it could not cause disease.

“We were scared, of course,” said Dr. Naldini, who was working on the virus in the laboratory of Dr. Inder M. Verma at the Salk Institute. But he reasoned that if he could remove enough H.I.V. genes to make it safe, the modified virus could work.

It solved the efficiency problem — the modified AIDS virus added genes to 15 percent of cells. And researchers believe it solved the cancer problem. The virus had less chance of turning on genes that could lead to cancer.

Ms. Salzman’s son had a bone marrow transplant. Her sister’s older boy died of ALD, and her younger son underwent a bone marrow transplant but had complications from the procedure and now, successfully treated for ALD, is in a wheelchair from side effects of the transplant.

Dr. Aubourg has also treated a third boy with ALD, and he is doing well, but it is too soon to know if his disease has been arrested.

“We have to be cautious — very, very cautious,” Dr. Aubourg said. “But this is the first time that a very serious disease of the brain, a lethal disease of the brain, has been treated with success by gene therapy.”

Here is the complete New York Times article and ScienceNow article.