Please use this identifier to cite or link to this item: doi:10.22028/D291-32970
Title: Cellular Targeting of Oligonucleotides by Conjugation with Small Molecules
Author(s): Hawner, Manuel
Ducho, Christian
Language: English
Title: Molecules
Volume: 25
Issue: 24
Publisher/Platform: MDPI
Year of Publication: 2020
Free key words: oligonucleotides
small molecules
cellular targeting
DDC notations: 500 Science
600 Technology
610 Medicine and health
Publikation type: Journal Article
Abstract: Drug candidates derived from oligonucleotides (ON) are receiving increased attention that is supported by the clinical approval of several ON drugs. Such therapeutic ON are designed to alter the expression levels of specific disease-related proteins, e.g., by displaying antigene, antisense, and RNA interference mechanisms. However, the high polarity of the polyanionic ON and their relatively rapid nuclease-mediated cleavage represent two major pharmacokinetic hurdles for their application in vivo. This has led to a range of non-natural modifications of ON structures that are routinely applied in the design of therapeutic ON. The polyanionic architecture of ON often hampers their penetration of target cells or tissues, and ON usually show no inherent specificity for certain cell types. These limitations can be overcome by conjugation of ON with molecular entities mediating cellular ‘targeting’, i.e., enhanced accumulation at and/or penetration of a specific cell type. In this context, the use of small molecules as targeting units appears particularly attractive and promising. This review provides an overview of advances in the emerging field of cellular targeting of ON via their conjugation with small-molecule targeting structures.
DOI of the first publication: 10.3390/molecules25245963
Link to this record: urn:nbn:de:bsz:291--ds-329702
ISSN: 1420-3049
Date of registration: 22-Jan-2021
Faculty: NT - Naturwissenschaftlich- Technische Fakultät
Department: NT - Pharmazie
Professorship: NT - Prof. Dr. Christian Ducho
Collections:SciDok - Der Wissenschaftsserver der Universität des Saarlandes

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