Journey of siRNA: Clinical Developments and Targeted Delivery

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 signifi...

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Published in	Nucleic acid therapeutics Vol. 28; no. 4; pp. 29 - 224
Main Authors	Nikam, Rahul R., Gore, Kiran R.
Format	Journal Article
Language	English
Published	United States Mary Ann Liebert, Inc 01.08.2018
Subjects	Biological evolution Cancer Chemical compounds Clinical trials Conjugation Drug development Enzymes Gene expression Gene silencing Glaucoma Hypertension Infections Ligands Macular degeneration Medical research Pain Peptides Pharmaceuticals Pharmacology Proteins Review Reviews Ribonucleic acid RNA RNA-mediated interference siRNA Vascular endothelial growth factor Mumbai India India clinical trial gene silencing targeted delivery siRNA RNA interference GalNAc
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Abstract	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.
AbstractList	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.
Author	Gore, Kiran R. Nikam, Rahul R.
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BackLink	https://www.ncbi.nlm.nih.gov/pubmed/29584585$$D View this record in MEDLINE/PubMed
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Snippet	Since the evolutionary discovery of RNA interference and its utilization for gene knockdown in mammalian cell, a remarkable progress has been achieved in small...
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SubjectTerms	Biological evolution Cancer Chemical compounds Clinical trials Conjugation Drug development Enzymes Gene expression Gene silencing Glaucoma Hypertension Infections Ligands Macular degeneration Medical research Pain Peptides Pharmaceuticals Pharmacology Proteins Review Reviews Ribonucleic acid RNA RNA-mediated interference siRNA Vascular endothelial growth factor
Title	Journey of siRNA: Clinical Developments and Targeted Delivery
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