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Critical Reviews™ in Therapeutic Drug Carrier Systems
Factor de Impacto: 2.9 Factor de Impacto de 5 años: 3.72 SJR: 0.736 SNIP: 0.551 CiteScore™: 2.43

ISSN Imprimir: 0743-4863
ISSN En Línea: 2162-660X

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Critical Reviews™ in Therapeutic Drug Carrier Systems

DOI: 10.1615/CritRevTherDrugCarrierSyst.v17.i4.30
48 pages

Targeted Gene Delivery: A Two-Pronged Approach

Khursheed Anwer
Expression Genetics, Inc., Huntsville, Alabama; and Valentis, Inc., 8301 New Trails Drive, The Woodlands, TX, USA
Austin Bailey
Valentis, Inc., 8301 New Trails Drive, The Woodlands, TX
Sean M. Sullivan
Valentis, Inc., 8301 New Trails Drive, The Woodlands, TX


The success of gene therapy relies on the ability of gene delivery systems to selectively deliver therapeutic genes to a sufficient number of target cells yielding expression levels that impact the diseased state. The gene delivery systems can be divided into two classes: viral and non-viral (or plasmid DNA-based). The present gene delivery technology being used in climes today can be considered first generation, in that they possess the ability to transfect or infect target cells through their inherent chemical, biochemical, and molecular biological properties. Relying on these sole properties, however, limits therapeutic applications. Expansion of therapeutic applications or increased effectiveness of current therapies can be achieved by increasing the number of cells and cell types susceptible to gene transfer. This can be achieved through physical targeting and molecular biological targeting. Physical targeting relies on the attachment to the delivery vehicle of ligands that bind to cell surface receptors unique to the target cells. Molecular biological targeting refers to selective expression of the therapeutic gene by the target cell through the use of selective promoters. Selective expression can be further achieved by the use of expression systems controlled by extrinsic induction molecules. This review will describe in detail the advances that have been made in each of these areas of gene targeting.