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Review
. 2005 Jul;12(14):1089-98.
doi: 10.1038/sj.gt.3302570.

Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors--design, biosafety, and production

P L Sinn  1 , S L Sauter, P B McCray Jr
Affiliations
Review

Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors--design, biosafety, and production

P L Sinn et al. Gene Ther. 2005 Jul.

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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