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Abstract
DNAzymes are DNA-based catalytic molecules that have potential use in a range of disorders where the targeted gene plays an important role in disease pathogenesis. DNAzymes are at a comparatively early developmental stage as alternatives to conventional therapies. The biological action of DNAzymes on target mRNA requires efficient delivery into target cells and this hurdle has hampered their broader use, particularly in systemic settings. DNAzymes have been delivered in naked form without a carrier or combined with agents such as polymers and liposomes. This article reviews these and other delivery approaches and offers perspectives on future methodologies for improved DNAzyme delivery and utility as novel drugs.
Keywords: Carrier, cationic, catalytic, DNAzymes, delivery, gene therapy, liposomes, nanoparticles, RNA cleavage, therapeutic.
Current Medicinal Chemistry
Title:DNAzyme Delivery Approaches in Biological Settings
Volume: 20 Issue: 28
Author(s): Cecilia W.S. Chan and Levon M. Khachigian
Affiliation:
Keywords: Carrier, cationic, catalytic, DNAzymes, delivery, gene therapy, liposomes, nanoparticles, RNA cleavage, therapeutic.
Abstract: DNAzymes are DNA-based catalytic molecules that have potential use in a range of disorders where the targeted gene plays an important role in disease pathogenesis. DNAzymes are at a comparatively early developmental stage as alternatives to conventional therapies. The biological action of DNAzymes on target mRNA requires efficient delivery into target cells and this hurdle has hampered their broader use, particularly in systemic settings. DNAzymes have been delivered in naked form without a carrier or combined with agents such as polymers and liposomes. This article reviews these and other delivery approaches and offers perspectives on future methodologies for improved DNAzyme delivery and utility as novel drugs.
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Cite this article as:
Chan W.S. Cecilia and Khachigian M. Levon, DNAzyme Delivery Approaches in Biological Settings, Current Medicinal Chemistry 2013; 20 (28) . https://dx.doi.org/10.2174/09298673113209990033
DOI https://dx.doi.org/10.2174/09298673113209990033 |
Print ISSN 0929-8673 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-533X |
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