Therapeutic face of RNAi: in vivo challenges

Abstract

Introduction: RNA interference is a sequence-specific gene silencing phenomenon in which small interfering RNAs (siRNAs) can trigger gene transcriptional and post-transcriptional silencing. This phenomenon represents an emerging therapeutic approach for in vivo studies by efficient delivery of specific synthetic siRNAs against diseases. Therefore, simultaneous development of synthetic siRNAs along with novel delivery techniques is considered as novel and interesting therapeutic challenges.

Areas covered: This review provides a basic explanation to siRNA signaling pathways and their therapeutic challenges. Here, we provide a comprehensive explanation to failed and successful trials and their in vivo challenges.

Expert opinion: Specific, efficient and targeted delivery of siRNAs is the major concern for their in vivo administrations. Also, anatomical barriers, drug stability and availability, immunoreactivity and existence of various delivery routes, different genetic backgrounds are major clinical challenges. However, successful administration of siRNA-based drugs is expected during foreseeable features. But, their systemic applications will depend on strong targeted drug delivery strategies.

Keywords:

• gene therapy
• small interfering RNA
• RNA interference
• small interfering RNA delivery

Acknowledgment

Authors thank Prof. Amir Maousavi (National Institute of Genetic Engineering and Biotechnology; NIGEB) for his encouragement and critical comments.

Declaration of interest

The authors were supported by Chemical Injuries Research Center, Baqiyatallah University of Medical Sciences,Tehran Iran. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Notes

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