Christopher Porada, PhD
Christopher Porada, PhD, Associate Professor
Dr. Christopher Porada received his Bachelor’s in Molecular Biology from Colgate University in 1991 and his PhD in Cellular and Molecular Pharmacology and Physiology from the University of Nevada in 1998. After completing his PhD, he served as an Assistant and then Associate Professor in the Department of Animal Biotechnology at the University of Nevada, Reno. He has authored over 40 scientific publications and reviews, has written chapters in several books, and served as an Associate Editor for Experimental Hematology. He is a member of several international societies including the American Society of Hematology, the International Society of Experimental Hematology, and the American Society of Gene and Cell Therapy, and regularly reviews for several international journals focused on gene therapy, gene and drug delivery, stem cell biology, and stem cell transplantation. Dr. Porada joined the faculty at WFIRM in 2011.
SYNOPSIS OF AREA OF INTEREST:
Dr. Porada’s research is broadly focused on the development of safer, more cost-effective treatments that could offer a permanent cure for a variety of genetic disorders. Specifically, he is developing novel gene delivery systems that precisely target individual cell types within the body and, is exploring the use of adult stem cells as vehicles for delivering therapeutic genes to specific sites of disease/injury.
DETAILED AREA OF INTEREST:
Gene therapy promises to offer a precise means of permanently curing a wide range of inherited and acquired diseases, once it has been fully optimized. Current methods rely on splicing the desired genetic material into a suitable virus and then harnessing the innate ability of the virus to deliver the genetic payload to the appropriate cells within the patient. Vectors based upon various viruses, in particular the retroviruses, have been used successfully to provide long-term correction in numerous animal models and in a limited number of human clinical trials. Despite these successes, however, existing vectors have often proven relatively inefficient at delivering genes to desirable targets such as hematopoietic stem cells, and they harbor certain risks, by virtue of their being derived from viruses that permanently integrate into the host cell genome. Further complicating treatment with gene therapy is the barrier posed by the recipient’s immune system, which frequently recognizes not only the viral-based gene delivery vehicle, but also the potentially curative gene product, as a foreign entity to be eliminated. We are currently focused on developing novel means of safely and efficiently performing gene therapy early in life, including before birth, thus side-stepping the immune hurdles which are present later in life and correcting the genetic lesion prior to the onset of disease and resultant tissue damage. Approaches being explored are: 1) the development of gene delivery systems that allow highly specific targeting of a single cell type following direct in vivo administration; 2) selection of appropriate target cells to ensure reliable induction of immunologic tolerance to the therapeutic transgene product; 3) the development of novel hybrid vectors which allow long-term gene correction in the absence of genomic integration; and 4) using genetically-modified hematopoietic and mesenchymal stem cells as gene delivery vehicles. Following development and optimization, hemophilia A is being used as a paradigm disease for proof-of-principle testing of these technologies.