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  • Title: Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors--design, biosafety, and production.
    Author: Sinn PL, Sauter SL, McCray PB.
    Journal: Gene Ther; 2005 Jul; 12(14):1089-98. PubMed ID: 16003340.
    Abstract:
    Replication defective vectors derived from simple retroviruses or the more complex genomes of lentiviruses continue to offer the advantages of long-term expression, cell and tissue specific tropism, and large packaging capacity for the delivery of therapeutic genes. The occurrence of adverse events caused by insertional mutagenesis in three patients in a gene therapy trial for X-linked SCID emphasizes the potential for problems in translating this approach to the clinic. Several genome-wide studies of retroviral integration are now providing novel insights into the integration site preferences of different vector classes. We review recent developments in vector design, integration, biosafety, and production.
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