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Design, mechanism, delivery and therapeutics of canonical and Dicer-substrate siRNA

Upon the discovery of RNA interference (RNAi), canonical small interfering RNA (siRNA) has been recognized to trigger sequence-specific gene silencing. Despite the benefits of siRNAs as potential new drugs, there are obstacles still to be overcome, including off-target effects and immune stimulation...

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Detalles Bibliográficos
Autores principales: Raja, Maria Abdul Ghafoor, Katas, Haliza, Amjad, Muhammad Wahab
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Shenyang Pharmaceutical University 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7032099/
https://www.ncbi.nlm.nih.gov/pubmed/32104477
http://dx.doi.org/10.1016/j.ajps.2018.12.005
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author Raja, Maria Abdul Ghafoor
Katas, Haliza
Amjad, Muhammad Wahab
author_facet Raja, Maria Abdul Ghafoor
Katas, Haliza
Amjad, Muhammad Wahab
author_sort Raja, Maria Abdul Ghafoor
collection PubMed
description Upon the discovery of RNA interference (RNAi), canonical small interfering RNA (siRNA) has been recognized to trigger sequence-specific gene silencing. Despite the benefits of siRNAs as potential new drugs, there are obstacles still to be overcome, including off-target effects and immune stimulation. More recently, Dicer substrate siRNA (DsiRNA) has been introduced as an alternative to siRNA. Similarly, it also is proving to be potent and target-specific, while rendering less immune stimulation. DsiRNA is 25–30 nucleotides in length, and is further cleaved and processed by the Dicer enzyme. As with siRNA, it is crucial to design and develop a stable, safe, and efficient system for the delivery of DsiRNA into the cytoplasm of targeted cells. Several polymeric nanoparticle systems have been well established to load DsiRNA for in vitro and in vivo delivery, thereby overcoming a major hurdle in the therapeutic uses of DsiRNA. The present review focuses on a comparison of siRNA and DsiRNA on the basis of their design, mechanism, in vitro and in vivo delivery, and therapeutics.
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spelling pubmed-70320992020-02-26 Design, mechanism, delivery and therapeutics of canonical and Dicer-substrate siRNA Raja, Maria Abdul Ghafoor Katas, Haliza Amjad, Muhammad Wahab Asian J Pharm Sci Review Article Upon the discovery of RNA interference (RNAi), canonical small interfering RNA (siRNA) has been recognized to trigger sequence-specific gene silencing. Despite the benefits of siRNAs as potential new drugs, there are obstacles still to be overcome, including off-target effects and immune stimulation. More recently, Dicer substrate siRNA (DsiRNA) has been introduced as an alternative to siRNA. Similarly, it also is proving to be potent and target-specific, while rendering less immune stimulation. DsiRNA is 25–30 nucleotides in length, and is further cleaved and processed by the Dicer enzyme. As with siRNA, it is crucial to design and develop a stable, safe, and efficient system for the delivery of DsiRNA into the cytoplasm of targeted cells. Several polymeric nanoparticle systems have been well established to load DsiRNA for in vitro and in vivo delivery, thereby overcoming a major hurdle in the therapeutic uses of DsiRNA. The present review focuses on a comparison of siRNA and DsiRNA on the basis of their design, mechanism, in vitro and in vivo delivery, and therapeutics. Shenyang Pharmaceutical University 2019-09 2019-02-13 /pmc/articles/PMC7032099/ /pubmed/32104477 http://dx.doi.org/10.1016/j.ajps.2018.12.005 Text en © 2019 Shenyang Pharmaceutical University. Published by Elsevier B.V. http://creativecommons.org/licenses/by-nc-nd/4.0/ This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Review Article
Raja, Maria Abdul Ghafoor
Katas, Haliza
Amjad, Muhammad Wahab
Design, mechanism, delivery and therapeutics of canonical and Dicer-substrate siRNA
title Design, mechanism, delivery and therapeutics of canonical and Dicer-substrate siRNA
title_full Design, mechanism, delivery and therapeutics of canonical and Dicer-substrate siRNA
title_fullStr Design, mechanism, delivery and therapeutics of canonical and Dicer-substrate siRNA
title_full_unstemmed Design, mechanism, delivery and therapeutics of canonical and Dicer-substrate siRNA
title_short Design, mechanism, delivery and therapeutics of canonical and Dicer-substrate siRNA
title_sort design, mechanism, delivery and therapeutics of canonical and dicer-substrate sirna
topic Review Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7032099/
https://www.ncbi.nlm.nih.gov/pubmed/32104477
http://dx.doi.org/10.1016/j.ajps.2018.12.005
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