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to Section One | to Arts & Entertainment
posted Friday, July 25 2014 - Volume 42 Issue 30
We can remove HIV from individual cells, researchers say
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We can remove HIV from individual cells, researchers say

by Mike Andrew - SGN Staff Writer

HIV can be eliminated from human cells using gene therapy, according to a new study by Temple University researchers.

The study was published July 21 in the Proceedings of the National Academy of Sciences.

Most current HIV treatments target the means by which HIV attaches itself to healthy host cells and then replicates itself. The Temple team, on the other hand, decided to try removing HIV from the infected cells.

'Since HIV-1 is never cleared by the immune system, removal of the virus is required in order to cure the disease,' lead researcher Dr. Kamel Khalili explained.

Khalili's team targeted the HIV genome using a strand of what is called 'guide RNA (gRNA)' plus an enzyme called a 'nuclease' that can excise the HIV DNA from the host cell. After that, the cell's gene repair machinery takes over, in effect splicing the loose ends of the genome back together, resulting in virus-free cells.

Researchers based the two-part approach on a system that evolved as a bacterial defense mechanism to protect against infection, Dr. Khalili said.

The therapy was successful in several cell types that can harbor HIV-1, including microglia and macrophages, as well as in T-lymphocytes.

'T-cells and monocytic cells are the main cell types infected by HIV-1, so they are the most important targets for this technology,' Khalili noted.

Highly active antiretroviral therapy (HAART) has been effective in controlling the virus in HIV-positive patients, but any interruption in treatment allows the HIV to reassert itself. Even when HIV replication is well controlled with antiretrovirals, the lingering HIV presence can have serious health consequences.

'The low level replication of HIV-1 makes patients more likely to suffer from diseases usually associated with aging,' Khalili said.

These include cardiomyopathy - a weakening of the heart muscle - bone disease, kidney disease, and neurocognitive disorders.

'These problems are often exacerbated by the toxic drugs that must be taken to control the virus,' Dr. Khalili added.

Controlled tests with the gene therapy method have proven to be successful so far, but Khalili says that his team still faces a number of significant challenges before the treatment is ready for patient trials.

To be completely effective the molecular agents responsible for disabling HIV's replication process would have to be delivered to every cell in a patient's body. Another difficulty is that HIV-1, the strain of HIV that the treatment has proved effective against, is prone to mutation, further complicating the therapy's effectiveness outside of controlled environments.

'We are working on a number of strategies so we can take the construct into preclinical studies,' Khalili said. 'We want to eradicate every single copy of HIV-1 from the patient. That will cure AIDS. I think this technology is the way we can do it.'

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