Abstract
Retroviral vectors are one of the most promising vehicles for the delivery of therapeutic genes in human gene therapy protocols. Retroviral-mediated gene transfer currently being used in human clinical trials is based upon ex vivo transduction of target cells. The ability to target the delivery and expression of therapeutic genes in vivo using retroviral vectors is a prerequisite for widespread and routine use in the clinic and will be of great importance for the safe and successful treatment of certain genetic disorders as well as tumors and viral infections. A number of approaches have been taken to develop retroviral vectors that are able to target particular cell types both at the level of the transduction event and at the level of expression. Using various combinations of the restrictive features reviewed in this article, it should be possible to achieve definitive targeting of genes transduced by retroviral vectors.
Overview summary
Targeting of the transfer and expression of genes will be essential in practical clinical gene therapy. A number of mechanisms are discussed by which such a controlled and limited expression of genes can be achieved. The use of one or more of these restrictive features should lead to safer vectors that will only transduce or be expressed in the tissue or organ to which the therapeutic gene product encoded within the vector is directed.
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