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.

Original languageEnglish
Pages (from-to)709-725
Number of pages17
JournalExpert Opinion on Drug Discovery
Volume13
Issue number8
DOIs
Publication statusPublished - 03-08-2018

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Small Interfering RNA
RNA Interference
Therapeutics
Expert Testimony
Endocytosis
Base Pairing
Cytosol
Messenger RNA
Serum
Pharmaceutical Preparations

All Science Journal Classification (ASJC) codes

  • Drug Discovery

Cite this

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title = "Strategies for improving the specificity of siRNAs for enhanced therapeutic potential",
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.",
author = "Gatta, {Aditya Kiran} and Hariharapura, {Raghu Chandrashekhar} and Nayanabhirama Udupa and Reddy, {Meka Sreenivasa} and Josyula, {Venkata Rao}",
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Strategies for improving the specificity of siRNAs for enhanced therapeutic potential. / Gatta, Aditya Kiran; Hariharapura, Raghu Chandrashekhar; Udupa, Nayanabhirama; Reddy, Meka Sreenivasa; Josyula, Venkata Rao.

In: Expert Opinion on Drug Discovery, Vol. 13, No. 8, 03.08.2018, p. 709-725.

Research output: Contribution to journalReview article

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AU - Gatta, Aditya Kiran

AU - Hariharapura, Raghu Chandrashekhar

AU - Udupa, Nayanabhirama

AU - Reddy, Meka Sreenivasa

AU - Josyula, Venkata Rao

PY - 2018/8/3

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N2 - 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.

AB - 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.

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