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CC BY V4.0, Open Access
Journal article
DNAzymes: Expanding the Potential of Nucleic Acid Therapeutics
Nucleic acid therapeutics, Vol.33(3), pp.178-192
2023
PMID: 37093127
Abstract
Nucleic acids drugs have been proven in the clinic as a powerful modality to treat inherited and acquired diseases. However, key challenges including drug stability, renal clearance, cellular uptake, and movement across biological barriers (foremost the blood–brain barrier) limit the translation and clinical efficacy of nucleic acid–based therapies, both systemically and in the central nervous system. In this study we provide an overview of an emerging class of nucleic acid therapeutic, called DNAzymes. In particular, we review the use of chemical modifications and carrier molecules for the stabilization and/or delivery of DNAzymes in cell and animal models. Although this review focuses on DNAzymes, the strategies described are broadly applicable to most nucleic acid technologies. This review should serve as a general guide for selecting chemical modifications to improve the therapeutic performance of DNAzymes.
Details
- Title
- DNAzymes: Expanding the Potential of Nucleic Acid Therapeutics
- Authors/Creators
- Leon M. Larcher - Murdoch UniversityIanthe L. Pitout - Murdoch University, Centre for Molecular Medicine and Innovative TherapeuticsNiall P. Keegan - Murdoch UniversityRakesh N. Veedu - Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Perth, Australia. Precision Nucleic Acid Therapeutics, Perron Institute for Neurological and Translational Science, Perth, AustraliaSue Fletcher - Murdoch University, Health Futures Institute
- Publication Details
- Nucleic acid therapeutics, Vol.33(3), pp.178-192
- Publisher
- Mary Ann Liebert, Inc.
- Identifiers
- 991005585959007891
- Copyright
- © 2023 Leon M. Larcher et al
- Murdoch Affiliation
- Centre for Molecular Medicine and Innovative Therapeutics
- Language
- English
- Resource Type
- Journal article
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