Gene transfer strategies for metabolic diseases.
Recent advances in molecular genetics and biotechnology will revolutionize the practice of medicine and surgery during the next decade. Surgeons must understand and begin to use the new technologies in the study of surgical biology and patient care. A brief overview of the principles of gene transfer and how this technology is used for treating metabolic disease is presented. Two examples of how recombinant viruses (vectors) can be used to study fundamental biology in the liver and pancreas are given. Familial hypercholesterolemia is used as a paradigm for a clinical gene transfer study and underscores the importance of surgeons in the development of novel therapeutic strategies.
Raper
Steven E
SE
Harrison Department of Surgical Research, University of Pennsylvania School of Medicine, 607 Biomedical Research Building II/III, 422 Curie Boulevard, Philadelphia, Pennsylvania 19104, USA. seraper@mail.med.upenn.edu
McClane
Steven J
SJ
D000256:Adenoviridae / Q000235:genetics; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000628:therapy*; D007150:Immunohistochemistry; D011519:Proto-Oncogenes / Q000235:genetics*