Journey of siRNA: Clinical Developments and Targeted Delivery
| Autor: | Rahul R. Nikam, Kiran R. Gore |
|---|---|
| Rok vydání: | 2018 |
| Předmět: |
0301 basic medicine
Drug Small interfering RNA media_common.quotation_subject Computational biology Biochemistry 03 medical and health sciences RNA interference Mammalian cell Drug Discovery Genetics Humans Gene silencing Medicine Gene Silencing RNA Small Interfering Molecular Biology media_common Gene knockdown business.industry Gene Transfer Techniques Genetic Therapy Clinical trial 030104 developmental biology Molecular Medicine RNA Interference business |
| Zdroj: | Nucleic Acid Therapeutics. 28:209-224 |
| ISSN: | 2159-3345 2159-3337 |
| DOI: | 10.1089/nat.2017.0715 |
| Popis: | Since the evolutionary discovery of RNA interference and its utilization for gene knockdown in mammalian cell, a remarkable progress has been achieved in small interfering RNA (siRNA) therapeutics. siRNA is a promising tool, utilized as therapeutic agent against various diseases. Despite its significant potential benefits, safe, efficient, and target oriented delivery of siRNA is one of the major challenges in siRNA therapeutics. This review covers major achievements in clinical trials and targeted delivery of siRNAs using various targeting ligand-receptor pair. Local and systemically administered siRNA drug candidates at various phases in clinical trials are described in this review. This review also provides a deep insight in development of targeted delivery of siRNA. Various targeting ligand-siRNA pair with complexation and conjugation approaches are discussed in this review. This will help to achieve further optimization and development in targeted delivery of siRNAs to achieve higher gene silencing efficiency with lowest siRNA dose availability. |
| Databáze: | OpenAIRE |
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