Clinical Chemistry
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Clinical Chemistry 55: 609-610, 2009. First published February 20, 2009; 10.1373/clinchem.2008.113019
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(Clinical Chemistry. 2009;55:609-610.)
© 2009 American Association for Clinical Chemistry, Inc.

Perspective

A New Tool for Oligonulceotide Import into Cells

Daniel C. Leslie1 and James P. Landers1,2,3,a

Departments of1 Chemistry; 2 Mechanical Engineering; and 3 Pathology, University of Virginia, Charlottesville, VA.

aAddress correspondence to this author at: Department of Chemistry, University of Virginia, McCormick Road, P.O. Box 400319, Charlottesville, VA 22904. Fax 434-243-8852; e-mail landers@virginia.edu.

The first 20% of the full text of this article appears below.

The field of gene therapy potentially offers physicians an entirely new set of armaments with which to battle disease. The approach, radical in concept but powerful in its potential effect, generically aims to correct defective genes responsible for disease development by inserting the normal gene into a nonspecific location in the genome or by swapping out the abnormal gene through homologous recombination. Perhaps more tangible, near-term approaches are those that look to either repair the abnormal gene via selective reverse mutation or "knockdown" the expression of the mutated gene. Delivery of oligonucleotides, whether as entire genes or shorter antisense strands, remains one of the most significant challenges in the field. The linchpin to success is the ability to efficiently and selectively insert oligonucleotides into the desired cells. Current efforts in the application of oligonucleotide delivery in this and other arenas are restricted by the relatively low efficiency of methods used for oligonucleotide transfer. In addition, potential hazards exist with any method that introduces foreign biological material into the body—toxicity issues as well as the potential for mounting an immune response against a specific gene delivery vehicle complicate the challenge.

It is against this backdrop that a series of papers out of the Mirkin . . . [Full Text of this Article]


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Copyright © 2009 by the American Association for Clinical Chemistry.