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UCLA researchers develop genetically engineered stem cells to fight HIV in mice

By Alexander Baklajian

April 19, 2012 12:39 a.m.

A team of UCLA researchers has found a genetic engineering technique that suppresses the HIV virus in mice, an encouraging step toward potentially fighting the disease in humans, researchers said.

The study, led by Scott Kitchen, a member of the UCLA AIDS Institute and assistant professor at the David Geffen School of Medicine, draws upon previous UCLA research findings. It was published last week in the epidemiology journal PLoS Pathogens and funded in part by the UCLA Center for AIDS Research.

Researchers examined the effectiveness of genetically engineered “killer” T cells, which are capable of fighting off disease, at combating the HIV virus in a mouse. The team used a “humanized” mouse engineered to have a human immune system. In the “humanized” mouse, the disease progressed similarly to its progression in humans, making it a reliable tool for the study and providing powerful predictive value for the therapy in humans, Kitchen said. “It’s a major advance and a step closer in demonstrating the potential use of this in people,” he said.

A few months ago, the team introduced a population of the engineered T cells into a mouse so they could develop and grow into a human immune system, Kitchen said. The researchers then conducted blood and organ tests at the second and sixth weeks, finding a decrease in the HIV levels and an increase in the cells HIV typically kills, according to the journal article.

The findings could theoretically be used to support a clinical trial in humans, said Jerome Zack, associate director at the UCLA AIDS Institute and co-author of the study.

A benefit genetic engineering is that it opens the field to therapeutic HIV treatments, and that it can be extended to potentially treat other diseases such as cancer, said co-author Arumugam Balamurugan.

In 2009, the lead scientists from the most recent study showed that human blood stem cells in mice could be genetically engineered to grow large quantities of “killer” T cells, As a result of their genetic engineering, these T cells grew to a large population and targeted HIV-infected cells in the mice.

“We had the idea that we could take the elements of immune response (the T cells) that are successful in suppressing HIV in infected people to see if it was possible to identify a receptor specific to HIV,” Kitchen said, referring to the team’s research in 2009.

Though advances have been made in the fight against HIV, an estimated 50,000 new cases are diagnosed in the United States each year, and there are more than 33 million people living with the disease worldwide, according to the National Institutes of Health. Factors that make the disease difficult to fight include its rapid rate of spread and lack of preventative measures. The findings could lead to more comprehensive methods of fighting the disease and eventually to a clinical trial in humans, Kitchen said.

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