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Updated: Jan 29, 2026

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
Published on: October 29, 2019
Topical and transdermal delivery of antisense oligonucleotides
1Department of Biological Systems Engineering, University of Nebraska-Lincoln, 68583-0726, USA. rbrand1@unl.edu
Abstract:
Antisense oligonucleotides have great potential as therapeutic agents. As a result, a variety of chemistries have been developed to improve the efficacy of these molecules without compromising their specificity. Because the skin is such a large organ with extensive accessibility, it is a natural target for drug delivery. It is, however, an effective barrier, so physical and chemical methods to improve drug penetration have been developed. These enhancement techniques can be combined with modified oligonucleotide chemistries to provide sufficient levels of antisense activity either within the skin or systemically. This review will describe in vitro and in vivo experiments that demonstrate the potential of antisense oligonucleotides to treat both dermal and systemic disorders.
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