Therapeutic face of RNAi: in vivo challenges. (February 2015)

• Record Type: Journal Article
• Authors: Borna, Hojat; Imani, Saber; Iman, Maryam; Azimzadeh Jamalkandi, Sadegh
• Is Part Of: Expert opinion on biological therapy. Volume 15:Number 2(2015:Feb.)
• Page Start: 269
• Page End: 285
• Publication Date: 2015-02
• Subjects: Gene therapy -- Periodicals; Protein drugs -- Periodicals; Peptide drugs -- Periodicals; Immunotherapy -- Periodicals; Drug delivery systems -- Periodicals; 615.5
• DOI: 10.1517/14712598.2015.983070
• Languages: English
• ISSNs: 1471-2598

Abstract

<abstract> <title> <x xml:space="preserve">Abstract</x> </title> <p> <bold> <italic>Introduction:</italic> </bold> 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 <italic>in vivo</italic> 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.</p> <p> <bold> <italic>Areas covered:</italic> </bold> 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 <italic>in vivo</italic> challenges.</p> <p> <bold> <italic>Expert opinion:</italic> </bold> Specific, efficient and targeted delivery of siRNAs is the major concern for their <italic>in vivo</italic> 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.</p> </abstract>