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Critical Reviews™ in Therapeutic Drug Carrier Systems
Импакт фактор: 2.9 5-летний Импакт фактор: 3.72 SJR: 0.736 SNIP: 0.551 CiteScore™: 2.43

ISSN Печать: 0743-4863
ISSN Онлайн: 2162-660X

Выпуски:
Том 36, 2019 Том 35, 2018 Том 34, 2017 Том 33, 2016 Том 32, 2015 Том 31, 2014 Том 30, 2013 Том 29, 2012 Том 28, 2011 Том 27, 2010 Том 26, 2009 Том 25, 2008 Том 24, 2007 Том 23, 2006 Том 22, 2005 Том 21, 2004 Том 20, 2003 Том 19, 2002 Том 18, 2001 Том 17, 2000 Том 16, 1999 Том 15, 1998 Том 14, 1997 Том 13, 1996 Том 12, 1995

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

Краткое описание

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.


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