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Title: RNA kot terapevtska tarča : priložnosti in omejitve v sodobni medicini Authors: ID Seliškar, Petra (Author) ID Hrast Rambaher, Martina (Author) ID Mravljak, Janez (Author) Files: PDF - Presentation file, download (999,86 KB) MD5: 54DC58E3EEC58E123F5E51454ADD2454   Language: Slovenian Typology: 1.02 - Review Article Organization: SFD - Slovenian Pharmaceutical Society Abstract: RNA je ključna biomolekula, ki poleg prenosa genetskih informacij uravnava celične procese in je pomembna terapevtska tarča. Njene regulatorne sposobnosti vključujejo utišanje genov ter usmerjanje epigenetskih modifikacij, kar omogoča razvoj zdravil, kot so protismiselni oligonukleotidi, male interferenčne RNA in aptameri. Protismiselni oligonukleotidi in male interfernčne RNA utišajo informacijsko RNA z vezavo na komplementarna zaporedja, medtem ko aptameri zavirajo interakcije med proteini. Kemijske modifikacije povečujejo stabilnost teh učinkovin. Določanje struktur RNA z metodami, kot so jedrska magnetna resonanca, krioelektronska mikroskopija in sekvenciranje, je ključno za razvoj terapij. Izzivi pri razvoju terapij RNA vključujejo dinamično naravo RNA, omejeno kemijsko raznolikost ter težave pri selektivnem ciljanju Keywords: aptameri, male molekule, RNA, siRNA, terapevtski oligonukleotidi Publication status: Published Publication version: Version of Record Publication date: 01.07.2025 Year of publishing: 2025 Number of pages: str. 184-192 Numbering: Letn. 76, št. 3 PID: 20.500.12556/DiRROS-27590  UDC: 577.113:615 ISSN on article: 0014-8229 COBISS.SI-ID: 245882627  Publication date in DiRROS: 13.02.2026 Views: 42 Downloads: 9 Metadata:    Cite this work Plain text BibTeX EndNote XML EndNote/Refer RIS ABNT ACM Ref AMA APA Chicago 17th Author-Date Harvard IEEE ISO 690 MLA Vancouver : Copy citation

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Record is a part of a journal

Title:	Farmacevtski vestnik : strokovno glasilo slovenske farmacije
Shortened title:	Farm. vestn.
Publisher:	Slovensko farmacevtsko društvo
ISSN:	0014-8229
COBISS.SI-ID:	3105794

Licences

License:	CC BY-NC 4.0, Creative Commons Attribution-NonCommercial 4.0 International
Link:	http://creativecommons.org/licenses/by-nc/4.0/
Description:	A creative commons license that bans commercial use, but the users don’t have to license their derivative works on the same terms.

Secondary language

Language:	English
Title:	RNA as therapeutic target : opportunities and limitations in modern medicine
Abstract:	RNA is a vital biomolecule that regulates cellular processes and therefore serves as a key therapeutic target. Its regulatory abilities include gene silencing and guiding epigenetic modifications, enabling the development of drugs like antisense oligonucleotides, small interference RNAs, and aptamers. Antisense oligonucleotides and small interference RNAs silence mRNA by binding to complementary sequences, while aptamers inhibit protein-protein interactions. Chemical modifications enhance the stability of these agents. Determining RNA structures using techniques such as nuclear magnetic resonance, cryogenic electron microscopy and sequencing is crucial for therapy development. Challenges in developing RNA-based therapies include RNA’s dynamic nature, limited chemical diversity, and difficulties in selective targeting.
Keywords:	aptamers, RNA, siRNA, small molecules, therapeutic oligonucleotides

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