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001-es BibID:	BIBFORM102994
035-os BibID:	(WoS)000845788200001 (Scopus)85137327522
Első szerző:	Bege Miklós (gyógyszerész)
Cím:	The Medicinal Chemistry of Artificial Nucleic Acids and Therapeutic Oligonucleotides / Miklós Bege, Anikó Borbás
Dátum:	2022
ISSN:	1424-8247
Megjegyzések:	Nucleic acids play a central role in human biology, making them suitable and attractive tools for therapeutic applications. While conventional drugs generally target proteins and induce transient therapeutic effects, nucleic acid medicines can achieve long-lasting or curative effects by targeting the genetic bases of diseases. However, native oligonucleotides are characterized by low in vivo stability due to nuclease sensitivity and unfavourable physicochemical properties due to their polyanionic nature, which are obstacles to their therapeutic use. A myriad of synthetic oligonucleotides have been prepared in the last few decades and it has been shown that proper chemical modifications to either the nucleobase, the ribofuranose unit or the phosphate backbone can protect the nucleic acids from degradation, enable efficient cellular uptake and target localization ensuring the efficiency of the oligonucleotide-based therapy. In this review, we present a summary of structure and properties of artificial nucleic acids containing nucleobase, sugar or backbone modifications, and provide an overview of the structure and mechanism of action of approved oligonucleotide drugs including gene silencing agents, aptamers and mRNA vaccines.
Tárgyszavak:	Természettudományok Kémiai tudományok idegen nyelvű folyóiratközlemény külföldi lapban folyóiratcikk oligonucleotides phosphorothioate phosphorodiamidate morpholino oligomers (PMOs) gene silencing antisense RNA interference aptamer splicing modulation mRNA vaccine
Megjelenés:	Pharmaceuticals. - 15 : 8 (2022), p. 1-39. -
További szerzők:	Borbás Anikó (1965-) (vegyész)
Pályázati támogatás:	K132870 OTKA GINOP-2.3.4-15-2020-00008 GINOP
Internet cím:	Szerző által megadott URL DOI Intézményi repozitóriumban (DEA) tárolt változat
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