Strategies for improving the Specificity of siRNAs for Enhanced Therapeutic Potential

Gatta, Aditya Kiran and Raghu, Chandrasekhar H and Udupa, N and Reddy, Sreenivasa M and Rao, Venkata Josyula (2018) Strategies for improving the Specificity of siRNAs for Enhanced Therapeutic Potential. Expert Opinion on Drug Discovery, 13 (8). pp. 709-725.

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Abstract

Introduction: RNA interference has become a tool of choice in the development of drugs in various therapeutic areas of Post Transcriptional Gene Silencing (PTGS). The critical element in developing successful RNAi therapeutics lies in designing small interfering RNA (siRNA) using an efficient algorithm satisfying the designing criteria. Further, translation of siRNA from bench-side to bedside needs an efficient delivery system and/or chemical modification.Areas covered: This review emphasizes the importance of dicer, the criteria for efficient siRNA design,the currently available algorithms and strategies to overcome off-target effects, immune stimulatory effects and endosomal trap.Expert opinion: Specificity and stability are the primary concerns for siRNA therapeutics. The design criteria and algorithms should be chosen rationally to have a siRNA sequence that binds to the corresponding mRNA as it happens in the Watson and Crick base pairing. However, it must evade a few more hurdles (Endocytosis, Serum stability etc.) to be functional in the cytosol.

Item Type: Article
Uncontrolled Keywords: RNA interference; siRNA; Strategies to overcome offtarget effects; Immune stimulatory effects and endosomal trap
Subjects: Pharmacy > MCOPS Manipal > Pharmaceutical Biotechnology
Research > Research Center - Health Sciences
Depositing User: KMC Library
Date Deposited: 21 Aug 2018 06:20
Last Modified: 21 Aug 2018 06:20
URI: http://eprints.manipal.edu/id/eprint/151791

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