Cell and Gene Therapy for HIV Cure Article Published by National Institutes of Health: https://pubmed.ncbi.nlm.nih.gov/29256135/... "Cell and gene therapy strategies are connected with human pathologies at a fundamental level, by delivering DNA and RNA molecules that could correct and/or ameliorate the underlying genetic factors of any illness...Today, HIV-based lentiviral vectors, along with many other gene delivery strategies, have been used to evaluate HIV cure approaches in cell culture, small and large animal models, and in patients..."
How does gene therapy work? "Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein."
Video Transcription:
I am Dave Pauza. I am the Chief Science Officer at American Gene Technologies in Rockville, Maryland. After my time receiving my PhD in Berkeley in 1981, I went to England for five years and I began my work on t-cell biology, thinking that would be a chance to study t-cell cancers; but at the same time back home, in the Bay Area, the scourge of HIV was on the rise and I was hearing these reports from friends and family about how terrible it has become and how rapidly this disease was spreading.
So I came back to the US in 1985 and I decided that would be my mission. It would be to work on HIV, to try to understand this disease and see what I could do to help. I began the HIV program at the Salk Institute before going to the University of Wisconsin in Madison and finally I was recruited to the Institute of Human Virology in Baltimore, Maryland in 2000 where I spent 17 years as Associate Director.
During that time, we really saw the impact of HIV on a, largely, minority population in Baltimore and realized that it was imperative to do something about this beyond just studying it. At the same time around 2013 I met Jeff Galvin, who was at that time running AGT and trying to work on lentivirus vector delivery to solve human disease and we went to work immediately trying to fill out the program and really trying to create a clinical path for drugs with priority on our cure for HIV and to take all steps necessary to make sure that we as a company were moving forward to attack something that we both felt deeply about and also realized that the conventional approaches had largely been exhausted and it was time for something new - and gene therapy was what was new.
We have this golden opportunity, now here at the real beginning of the age of genetic medicine. We have the opportunity to participate and be pioneers in really curing things. It's an endpoint for people who will take that cure therapy and actually become cured and never have to take their medicines again and never have to think of HIV again, but it's also a hope for everybody else that they'll want to see their doctors, get their testing, enroll in therapy and try to be a candidate for that same cure.
In addition to being a product, that the company is moving along in that conventional sense, it's also a mission to try to improve the awareness and improve the uptake of clinical care and help for people with HIV so that we can make a difference worldwide. This is a terrible, frightening and horrible disease. Once you have it, you think about it every day. The medications that people take are not like Aspirin; they are like chemotherapy and it’s a struggle to maintain their compliance and adherence to their drugs so they can stay healthy. We'd like to put a stop to that and bring the cure.
About Chief Science Officer C. David Pauza, Ph.D.:
C. David Pauza, Ph.D. is Chief Science Officer for American Gene Technologies and Professor of Medicine at the University of Maryland Medical School in Baltimore. Prior to joining AGT, Dr. Pauza was Associate Director for the university’s prestigious Institute of Human Virology and Co-Leader of the Greenebaum Cancer Center Program in Viral Oncology. He is an internationally recognized expert in human virology and viral diseases including HIV, arenaviruses, poxviruses and herpesviruses. He has published more than 150 scientific papers and holds 7 US patents.
