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Translational Medicine AAV Vectors for Infectious Diseases University of Pennsylvania Gene Therapy Team Finds Applications in HIV, Flu, and Biothreats James M. Wilson, M.D., Ph.D. The failure of the adenovirus Ad5 vaccine in the STEP study, halted in 2007, sparked renewed interest in developing antibody-based, rather than T-cell-based, vaccines against the AIDS virus. However, HIV evolves so rapidly that developing a vaccine formulation eliciting the necessary breadth of antibodies against the virus has proven diffcult. The challenge of an antibody-based approach is that the antibodies which form against HIV antigens are not broad enough to protect against the diversity of HIV isolates. Some laboratories have successfully cloned B cells, including their antibody genes, from HIV-infected patients whose disease had not progressed. They found that the SAVE $100 WITH KEYCODE J99! The 2009 H1N1 pandemic demonstrated that the time required to generate, manufacture, and distribute a traditional vaccine was unfeasible. Fotolia 5th Annual September 25-27, 2013 Seaport World Trade Center | Boston, MA Empowering Patients and Consumers with Advances in Genomics, Diagnostics and Personalized Healthcare Program Topics: • Whole Genome Debates • Translational Genomics • Clinical & Third-Generation Sequencing • Personal Genome Analysis & Interpretation • Empowering Patients: Companies & Technologies • Molecular Diagnostics & Point-of-Care • Investment & Funding Opportunities • Reimbursement Models • Five-Year Plan for Consumer Genomics • Data Analysis & Management • Ethics, Privacy & Regulation • Digital Health Tracking Apps The Consumer Genetics Conference (CGC) is a one-of-a-kind event that draws together a dynamic community of scientists, clinicians, technology innovators, and patients to discuss the burning issues around the analysis and delivery of genomics results as presented directly to patients and consumers. Over three days, attendees will hear about disruptive diagnostic technologies, cognitive barriers to patients (and medical professionals), ethical/ regulatory/privacy issues, the thorny issue of reimbursement, and the challenges of building relationships to realize the potential of personal genomics and individualized medicine. CGC provides an opportunity for all stakeholders to come together at one venue, share viewpoints and engage in an honest dialogue, and together learn how to move the elephant of change. Hear Keynotes from: Hugh Young Rienhoff, Jr., M.D., Director, MyDaughtersDNA.org Lead Sponsoring Publication: Spencer Wells, Ph.D., Explorer-in-Residence and Director, The Genographic Project, National Geographic Society Please use keycode J99 when registering 50 | ConsumerGeneticsConference.com September 1, 2013 | GENengnews.com | Genetic Engineering & Biotechnology News antibodies from these B cells bound to a portion of HIV that broadly neutralized against many HIV isolates. While the cloned antibodies could potentially prevent HIV if expressed in humans, this approach would require frequent reinjection, as antibodies degrade over time. The required regimen is also expensive and diffcult to adhere to and simply not possible in developing countries where an effective HIV vaccine is most needed. A few years back, the Bill & Melinda Gates Foundation approached the Gene Therapy Program at Penn about leveraging its experience in adeno-associated virus (AAV) vectors to develop a method for delivering antibodies against HIV. Philip R. Johnson, M.D., a professor at The Children's Hospital of Philadelphia, showed success with the approach in a study published in 2009 in Nature Medicine. Dr. Johnson's team created immunoadhesins designed to bind to simian immunodefciency virus (SIV) and block it from infecting cells. SIV-specifc immunoadhesin DNA was engineered into AAV carriers for injection in monkeys, where the DNA produced high systemic levels of the immunoadhesins. Most immunized monkeys injected with live SIV were completely protected from SIV infection, and all were protected from AIDS. Unimmunized monkeys were all infected by SIV, and two-thirds died of AIDS complications. The Gene Therapy Program was asked to assess the feasibility of this technique using a new generation of AAV vectors developed in my laboratory. Our initial work focused on James M. Wilson, M.D., Ph.D., is professor and director of the gene therapy program in the department of pathology and laboratory medicine, and professor in the department of internal medicine at the University of Pennsylvania. He is a consultant to ReGenX Holdings, and is a founder of, holds equity in, and receives a grant from affliates of ReGenX Holdings. In addition, he is an inventor on patents licensed to various biopharmaceutical companies, including affliates of ReGenX Holdings.