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 language | English |
|---|---|
| Pages (from-to) | 709-725 |
| Number of pages | 17 |
| Journal | Expert Opinion on Drug Discovery |
| Volume | 13 |
| Issue number | 8 |
| DOIs | |
| Publication status | Published - 03-08-2018 |
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All Science Journal Classification (ASJC) codes
- Drug Discovery
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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 journal › Review article
TY - JOUR
T1 - Strategies for improving the specificity of siRNAs for enhanced therapeutic potential
AU - Gatta, Aditya Kiran
AU - Hariharapura, Raghu Chandrashekhar
AU - Udupa, Nayanabhirama
AU - Reddy, Meka Sreenivasa
AU - Josyula, Venkata Rao
PY - 2018/8/3
Y1 - 2018/8/3
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.
UR - http://www.scopus.com/inward/record.url?scp=85050772280&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85050772280&partnerID=8YFLogxK
U2 - 10.1080/17460441.2018.1480607
DO - 10.1080/17460441.2018.1480607
M3 - Review article
VL - 13
SP - 709
EP - 725
JO - Expert Opinion on Drug Discovery
JF - Expert Opinion on Drug Discovery
SN - 1746-0441
IS - 8
ER -