May 09, 2015
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NIH awards $7.5 million to Penn Medicine for HIV gene therapy

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The NIH granted $7.5 million to researchers from the Perelman School of Medicine and the Penn Center for AIDS Research for the development of a novel HIV-gene therapy, according to a press release.

Penn Medicine researchers will partner with Sangamo BioSciences to combine the viral entry inhibitor, C34, with the HIV coreceptor, CXCR4. The fused synthetic molecule has the potential to block the HIV virus from entering CD4 T-cells, which would control the spread of HIV-1 without antiretroviral drug therapies, according to the release.

In a phase 1 trial, T-cells extracted from patients with HIV will be compromised and infused back into the patients to determine the molecule’s resistance and efficacy.

Previously, researchers replicated and induced the CCR5-delta-32 mutation, an HIV-resistant mutation effective in 1% of the general population, in 12 patients with HIV. The simulated mutation reduced surface protein expression of HIV coreceptor CCR5. This disrupted viral entry and decreased viral loads in patients not assigned ART, including one patient whose virus was no longer detectable.

"In this next trial, we hypothesize that individuals who receive engineered T-cells expressing the C34-CXCR4 fusion gene will experience a significant loss in viral load for a longer period of time because we are able to protect CD4 T-cells from different classes of HIV-1," James L. Riley, PhD, associate professor of microbiology at Perelman, said in the release. "This is an important component of the HIV gene therapy work here at Penn, as it will tell us how these approaches stack up against each other. We are ultimately trying to find effective, lasting ways to eliminate the need for lifelong [antiretroviral drug therapy] for HIV infected patients, and we believe these projects will help find the answers to some of the hard questions surrounding that goal."

The 5-year grant is funded through the NIH’s U19 Research Program, which is supported by the National Institute of Allergy and Infectious Disease, the National Heart, Lung and Blood Institute and the National Institute of Mental Health.