Abstract

The objective of this review is to consider the most significant advances that have been made in the past few years in the use of retroviral vectors for gene expression. Undoubtedly, one of the most significant of these has been the first sanctioned use, in May 1989, of such agents for human clinical studies. Significant progress has also been achieved in the in vivo expression of the adenosine deaminase (ADA) gene inserted into mouse bonemarrow cells and reintroduced via bone-marrow transplantation (BMT), in the retroviral-mediated expression of other genes of clinical relevance, and in retroviral vector technology itself, including construction of new types of vectors, attempts to increase the titers of retroviral supernatants, and some detailed safety studies particularly in primates. The next few years promise to yield substantial clinical evaluation of retroviral vectors for use not only in the correction of genetic defects, but also as a generalized drug-delivery system for protein therapeutics. In the latter application, genes expressed from retroviral vectors have the potential to solve some of the substantial problems associated with protein delivery using conventional delivery modalities.

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