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Chemically Modified Platforms for Better RNA Therapeutics.

Authors :
Shi, Yesi
Zhen, Xueyan
Zhang, Yiming
Li, Yongjiang
Koo, Seyoung
Saiding, Qimanguli
Kong, Na
Liu, Gang
Chen, Wei
Tao, Wei
Source :
Chemical Reviews. 2/14/2024, Vol. 124 Issue 3, p929-1033. 105p.
Publication Year :
2024

Abstract

RNA-based therapies have catalyzed a revolutionary transformation in the biomedical landscape, offering unprecedented potential in disease prevention and treatment. However, despite their remarkable achievements, these therapies encounter substantial challenges including low stability, susceptibility to degradation by nucleases, and a prominent negative charge, thereby hindering further development. Chemically modified platforms have emerged as a strategic innovation, focusing on precise alterations either on the RNA moieties or their associated delivery vectors. This comprehensive review delves into these platforms, underscoring their significance in augmenting the performance and translational prospects of RNA-based therapeutics. It encompasses an in-depth analysis of various chemically modified delivery platforms that have been instrumental in propelling RNA therapeutics toward clinical utility. Moreover, the review scrutinizes the rationale behind diverse chemical modification techniques aiming at optimizing the therapeutic efficacy of RNA molecules, thereby facilitating robust disease management. Recent empirical studies corroborating the efficacy enhancement of RNA therapeutics through chemical modifications are highlighted. Conclusively, we offer profound insights into the transformative impact of chemical modifications on RNA drugs and delineates prospective trajectories for their future development and clinical integration. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
00092665
Volume :
124
Issue :
3
Database :
Academic Search Index
Journal :
Chemical Reviews
Publication Type :
Academic Journal
Accession number :
175450323
Full Text :
https://doi.org/10.1021/acs.chemrev.3c00611