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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...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Shenyang Pharmaceutical University
2019
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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. |
format | Online Article Text |
id | pubmed-7032099 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | Shenyang Pharmaceutical University |
record_format | MEDLINE/PubMed |
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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