Chemically Modified Platforms for Better RNA Therapeutics

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.

Medienart:

E-Artikel

Erscheinungsjahr:

2024

Erschienen:

2024

Enthalten in:

Zur Gesamtaufnahme - volume:124

Enthalten in:

Chemical reviews - 124(2024), 3 vom: 14. Feb., Seite 929-1033

Sprache:

Englisch

Beteiligte Personen:

Shi, Yesi [VerfasserIn]
Zhen, Xueyan [VerfasserIn]
Zhang, Yiming [VerfasserIn]
Li, Yongjiang [VerfasserIn]
Koo, Seyoung [VerfasserIn]
Saiding, Qimanguli [VerfasserIn]
Kong, Na [VerfasserIn]
Liu, Gang [VerfasserIn]
Chen, Wei [VerfasserIn]
Tao, Wei [VerfasserIn]

Links:

Volltext

Themen:

63231-63-0
Journal Article
RNA
RNA, Small Interfering
Research Support, Non-U.S. Gov't
Review

Anmerkungen:

Date Completed 15.02.2024

Date Revised 26.03.2024

published: Print-Electronic

Citation Status MEDLINE

doi:

10.1021/acs.chemrev.3c00611

funding:

Förderinstitution / Projekttitel:

PPN (Katalog-ID):

NLM36775021X