Int J Med Sci 2004; 1(2):76-91. doi:10.7150/ijms.1.76
Gene Therapy: The Potential Applicability of Gene Transfer Technology to the Human Germline
School of Contemporary Sciences, University of Abertay, Dundee, DD1 1HG, United Kingdom
Smith KR. Gene Therapy: The Potential Applicability of Gene Transfer Technology to the Human Germline. Int J Med Sci 2004; 1(2):76-91. doi:10.7150/ijms.1.76. Available from http://www.medsci.org/v01p0076.htm
The theoretical possibility of applying gene transfer methodologies to the human germline is explored. Transgenic methods for genetically manipulating embryos may in principle be applied to humans. In particular, microinjection of retroviral vector appears to hold the greatest promise, with transgenic primates already obtained from this approach. Sperm-mediated gene transfer offers potentially the easiest route to the human germline, however the requisite methodology is presently underdeveloped. Nuclear transfer (cloning) offers an alternative approach to germline genetic modification, however there are major health concerns associated with current nuclear transfer methods. It is concluded that human germline gene therapy remains for all practical purposes a future possibility that must await significant and important advances in gene transfer technology.
Keywords: gene targeting, gene therapy, genetic modification, pronuclear microinjection, sperm-mediated gene transfer