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医药载体系统评论综述
影响因子: 2.9 5年影响因子: 3.72 SJR: 0.736 SNIP: 0.551 CiteScore™: 2.43

ISSN 打印: 0743-4863
ISSN 在线: 2162-660X

医药载体系统评论综述

DOI: 10.1615/CritRevTherDrugCarrierSyst.2014007943
pages 305-329

Small RNAs: The Qualified Candidates for Gene Manipulation in Diverse Clinical Pathologies

Indu Pal Kaur
University Institute of Pharmaceutical sciences, Panjab University, Chandigarh, India-160014
Kanwaljit Chopra
University Institute of Pharmaceutical Sciences, Panjab University, Chandigarh 160014, India
Parveen Rishi
Department of Microbiology, Basic Medical Sciences Block, Panjab University, Chandigarh, India
Sanjeev Puri
Department of Biotechnology, University Institute of Engineering & Technology, Panjab University, Chandigarh, India
Gaurav Sharma
University Institute of Pharmaceutical Sciences, Panjab University, Chandigarh 160014, India

ABSTRACT

In recent years, RNA interference (RNAi) has become a tool of choice to analyze and target the in vitro and in vivo function of mammalian genes. RNAi down-regulates gene expression by inducing enzyme-dependent degradation of targeted mRNA. This can be achieved using small double-stranded RNAs (dsRNAs), including small interfering RNAs (siRNAs), micro RNAs, and piwi RNAs. These active, small dsRNAs can regulate endogenous genes in both somatic and germ cells such that they can defend the genome from invasive nucleic acids. Extension of the concept of RNAi to preclinical studies indicates its probable application in the treatment of cancers, viral infections, arterial stenosis, and genetic disorders. Exciting results from ongoing clinical trials have raised the expectations of the scientific community for the use of RNAi in "real cures". Rational pharmaceutical design of these molecules can further open a Pandora's box of newer therapeutic options. However, efficient delivery of these small dsRNAs to target tissues or cells and their unanticipated nonspecific effects comprise a few important issues that still need to be addressed. In this review, we offer an overview of different small dsRNAs, their biogenesis, and their applicability in therapeutics. Current delivery strategies and formulation techniques to achieve the desired transfection capability and RNAi products in clinical trials are also discussed.