Getting to Know Gene Therapy: Terminology and Concepts
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Understanding key terminologies in hemophilia treatment is critical. Certain viruses work well as vectors to deliver a new gene to the proper location. These modified viruses infect the cell as part of the therapeutic process. The modification of these viruses prevents them from causing disease.
Vectors in gene therapy deliver DNA to the diseased cells using various carriers such as retroviruses and adenoviruses. Retroviruses, used for this purpose, also integrate their genetic material to include the new gene into the human cell. Efficacy vs. effectiveness is also crucial in gene therapy. Efficacy refers to a specific gene therapy intervention's performance under ideal but controlled circumstances. Effectiveness is the performance of the therapy in real-world conditions.
In Getting to Know Gene Therapy: Terminology and Concepts led by David Lillicrap, MD, PhD, you will discover a comprehensive educational resource designed by leading experts to help you stay up-to-date with the evolving science and clinical advancements in hemophilia research.
David Lillicrap, MD, FRCPC
Queen’s University - Kingston, Canada
David Lillicrap, MD, FRCPC, is a Professor in the Department of Pathology and Molecular Medicine at Queen’s University, Kingston, Canada. He is the recipient of a Senior Canada Research Chair in Molecular Hemostasis. In 2013, he was elected to the Fellowship of the Royal Society of Canada. Dr. Lillicrap is a member of the World Federation of Hemophilia’s (WFH) Medical Advisory Board and a past Chair of the WFH’s Research Committee. He is a past Chairman of the International Society on Thrombosis and Haemostasis’ (ISTH) Scientific and Standardization Committee and is a current member of the Council of ISTH. Between 2008-2014 he served as an Associate Editor of Blood, and is currently Co-Editor-in-Chief of the Journal of Thrombosis and Haemostasis. Dr. Lillicrap’s research interests are centered on molecular aspects of the hemostatic system, with a particular emphasis on the potential of molecular genetics and molecular biology to address questions relating to pathological hemostasis. The main focus of interest of his research group are the investigation of the immune response to FVIII, the development and evaluation of novel therapies for hemophilia A, and characterization of the biology and pathobiology of von Willebrand factor.
