Gene therapy: the potential applicability of gene transfer technology to the human germline

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    Abstract

    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.
    Original languageEnglish
    Pages (from-to)247-268
    Number of pages22
    JournalInternational Journal of Medical Sciences
    Volume34
    Issue number4
    DOIs
    Publication statusPublished - Jun 2004

    Keywords

    • Sperm-mediated gene therapy
    • Gene transfer
    • Genetic modification
    • Pronuclear microinjection
    • Gene targeting

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