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posted Friday, March 7 2014 - Volume 42 Issue 10
Gene therapy HIV treatment
Section One
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Gene therapy HIV treatment

by Mike Andrew - SGN Staff Writer

A new study published in the New England Journal of Medicine suggests that gene therapy can help HIV patients stop the progress of the disease by making T-cells permanently resistant to infection.

Researchers at the University of Pennsylvania observed that about 1% of humans are naturally resistant to HIV infection because they lack the specific protein that the virus attaches itself to. Their study aimed at altering the DNA of nonresistant T-cells to reproduce this genetic mutation, then reintroducing them into HIV patients.

Scientists hoped that the new method would produce HIV-resistance in their patients and stop the progress of the disease, and the results so far have been encouraging they said.

'This is a first - gene editing has not to date been used in a human trial [for HIV],' Prof. Bruce Levine, the director of the Clinical Cell and Vaccine Production Facility at the University of Pennsylvania, said:

'We've been able to use this technology in HIV and show it is safe and feasible, so it is an evolution in the treatment of HIV from daily antiretroviral therapy.'

First, millions of T-cells were harvested from the blood of the 12 patients in the study. The cells were then allowed to reproduce in the laboratory until researchers had billions of cells to use.

The team then edited the DNA inside the T-cells to give them the HIV-resistant mutation - known as CCR5-delta-32.

About 20% of the T-cells were successful modified, and these were then reintroduced into the HIV patients along with other, unmodified cells.

When patients were taken off their antiretroviral meds for four weeks, the number of unprotected T-cells still in the body fell dramatically, whereas the genetically modified T-cells seemed to be protected and could still be found in the blood several months later.

The finding raised hopes that genetic therapy could free HIV patients from the necessity of sticking to a daily meds regimen.

'The idea of modifying a T-cell to make it resistant and showing it is feasible and they survive - that's exciting in itself,' Prof. Sharon Lewin from Monash University in Australia, told BBC News:

'What most people are aiming for in HIV is a way you take treatment for a short period of time and that keeps the virus under control.'

She said drug treatment would not be replaced by this treatment, especially in the early stages of the infection, but it might lead to people eventually replacing drugs with gene therapy.

The University of Pennsylvania trial was designed to test only the safety and feasibility of the method, not whether it could replace drug treatment in the long term.

Meanwhile, the White House released a statement saying the National Institutes of Health (NIH) had endorsed gene therapy as a 'generally safe' drug-free alternative for the treatment of HIV/AIDS.

After describing the experimental process, the March 5 White House statement added that future testing will be needed to determine if the method is viable as a treatment.

'Future research will include evaluating this experimental treatment in more volunteers, as well as maximizing the frequency of CCR5 disruption by [genetic modification] and increasing the persistence of the genetically modified cells in the body to achieve a therapeutic effect,' the statement said.

The study was funded by Sangamo BioSciences, a pharmaceutical company.

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