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Scheideler, M. ; Vidakovic, I.* ; Prassl, R.*

Lipid nanocarriers for microRNA delivery.

Chem. Phys. Lipids 226, DOI: 10.1016/j.chemphyslip.2019.104837 (2020)
Postprint DOI Verlagsversion bestellen
Open Access Green
Non-coding RNAs (ncRNAs) like microRNAs (miRNAs) or small interference RNAs (siRNAs) with their power to selectively silence any gene of interest enable the targeting of so far 'undruggable' proteins and diseases. Such RNA molecules have gained much attention from biotech and pharmaceutical companies, which led to the first Food and Drug Administration (FDA) approved ncRNA therapeutic in 2018.However, the main barrier in clinical practice of ncRNAs is the lack of an effective delivery system that can protect the RNA molecules from nuclease degradation, deliver them to specific tissues and cell types, and release them into the cytoplasm of the targeted cells, all without inducing adverse effects.For that reason, drug delivery approaches, formulations, technologies and systems for transporting pharmacological ncRNA compounds to achieve a diagnostic or therapeutic effect in the human body are in demand.Here, we review the development of therapeutic lipid -based nanoparticles for delivery of miRNAs, one class of endogenous ncRNAs with specific regulatory functions. We outline challenges and opportunities for advanced miRNA-based therapies, and discuss the complexity associated with the delivery of functional miRNAs. Novel strategies are addressed how to deal with the most critical points in miRNA delivery, such as toxicity, specific targeting of disease sites, proper cellular uptake and endosomal escape of miRNAs. Current fields of application and various preclinical settings involving miRNA therapeutics are discussed, providing an outlook to future clinical approaches.Following the current trends and technological developments in nanomedicine exciting new delivery systems for ncRNA-based therapeutics can be expected in upcoming years.
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Publikationstyp Artikel: Journalartikel
Dokumenttyp Review
Schlagwörter Liposomes ; Lipoplexes ; Lipid Nanoparticles ; Oligonudeotides; In-vivo Delivery; Targeted Delivery; Cationic Lipids; Lipopolyplex Nanoparticles; Cancer-therapy; Sirna Delivery; Drug-delivery; Stem-cells; Pharmaceutical Nanocarriers; Antisense Oligonucleotides
ISSN (print) / ISBN 0009-3084
e-ISSN 0009-3084
Quellenangaben Band: 226 Heft: , Seiten: , Artikelnummer: , Supplement: ,
Verlag Elsevier
Verlagsort Elsevier House, Brookvale Plaza, East Park Shannon, Co, Clare, 00000, Ireland
Begutachtungsstatus Peer reviewed