The Department of Biological Sciences will be holding its 10th Annual Biological Sciences Symposium as part of the Petersheim Exposition on April 20, 2017. This event is open to all members of the university community and their invited guests. It will start with a student poster session at 3:30 p.m. in the McNulty Science Center Atrium and will feature keynote speaker Kamel Khalili, PhD, who is Professor and Chair of the Department of Neuroscience at the Lewis Katz School of Medicine at Temple University.
Dr. Khalili is an internationally renowned scientist. Using gene editing technology to successfully excise a segment of HIV-1 DNA – the virus responsible for AIDS from cells in in vitro, ex vivo and in vivo systems, Dr. Khalili's research is a critical step in the development of a potentially curative strategy for HIV infection. The new gene therapy technology can target copies of the HIV virus that lie in the genome, effectively suppressing infection. In addition to his position in the Department of Neuroscience, he is also Director for the Center of Neurovirology and Director for the Comprehenstive NeuroAIDS Center at Temple University. His keynote address is "Gene Editing Strategy for Cure of AIDS," and will be presented in McNulty Amphitheater Room 101 starting at 5:25pm.
"In a proof-of-concept study, we show that our gene editing technology can be effectively delivered to many organs of two small animal models and excise large fragments of viral DNA from the host cell genome," explained Dr. Khalili.
Current treatment for HIV infection uses a combination of antiretroviral drugs. This can effectively suppress HIV replication, but has no ability to eliminate HIV-1 from infected cells. When this course of treatment is interrupted, HIV replication rebounds. Patients are at risk for developing acquired immune deficiency syndrome, or AIDS.
Dr. Khalili's novel gene editing system, based on CRISPR/Cas9 technology, has the extraordinary ability to eliminate HIV-1 from infected cells in vitro with no adverse effect on the host cells. In ex vivo experiments using clinical specimens, including T-cells from patients infected with HIV that were expanded in culture, they showed that viral replication was significantly reduced following treatment with the gene editing system.
The clinical implications of the new research is far-reaching. The gene editing platform with further development is expected to eradicate HIV-1 DNA from patients. It is also highly flexible and potentially could be used in combination with existing antiretroviral drugs to further suppress viral RNA. It also could be adapted to target mutated strains of HIV-1.
The follow-up studies will allow Dr. Khalili's teams to monitor for effects of the treatment, its safety, and other important indices. With his continuous success in gene editing strategy, clinical trials are expected to happen within the next few years.