Gene Therapy Progress and Prospects: Development of improved lentiviral and retroviral vectors – design, biosafety, and production
Top Cited Papers
- 1 July 2005
- journal article
- review article
- Published by Springer Nature in Gene Therapy
- Vol. 12 (14) , 1089-1098
- https://doi.org/10.1038/sj.gt.3302570
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.Keywords
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