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Molecules
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Antisense Oligonucleotide Chemistry and Applications
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Antisense Oligonucleotide Chemistry and Applications

A special issue of Molecules (ISSN 1420-3049). This special issue belongs to the section "Bioorganic Chemistry".

Deadline for manuscript submissions: closed (20 June 2019) | Viewed by 25949

Special Issue Editor

Dr. Rakesh N. Veedu

E-Mail Website
Guest Editor
Centre for Comparative Genomics & Perron Institute for Neurological & Translational Science, Murodch University, Perth, Australia
Interests: antisense oligonucleotides; siRNA; aptamers; miRNA; DNAzymes; triplex forming oligonucleotides; chemically-modified oligonucleotides; oligonucleotide delivery
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Synthetic antisense oligonucleotide (AO)-based therapy has attracted significant interests in the last decade, and offers enormous potential as therapeutics to various diseases. To date, five AO drugs have been approved by the US Food and Drug Administration (FDA) for clinical use including Vitravene for cytomegalovirus retinitis; Kynamro for familial hypercholesterolemia, Exondys51 for the treatment of Duchenne muscular dystrophy, Spinraza for the treatment of spinal muscular atrophy and very recently Tegsedi for Hereditary Transthyretin Amyloidosis. As you all know from these clinical translations that the use of chemically-modified nucleotide monomers in constructing AOs is absolutely essential in developing successful drugs.

This special issue covers various aspects of antisense oligonucleotide synthesis chemistries, and their applications in molecular medicine in the form of authentic research and review articles.

Dr. Rakesh N. Veedu
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Molecules is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2700 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • Antisense Oligonucleotides
  • Antisense Therapy
  • Chemically-modified Antisense Oligonucleotides
  • Exon-skipping
  • Nucleic Acid Chemistry

Published Papers (3 papers)

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Research

11 pages, 1766 KiB  
Article
In Vitro Validation of Phosphorodiamidate Morpholino Oligomers
by May T. Aung-Htut, Craig S. McIntosh, Kristin A. West, Sue Fletcher and Steve D. Wilton
Molecules 2019, 24(16), 2922; https://doi.org/10.3390/molecules24162922 - 12 Aug 2019
Cited by 16 | Viewed by 4885
Abstract
One of the crucial aspects of screening antisense oligonucleotides destined for therapeutic application is confidence that the antisense oligomer is delivered efficiently into cultured cells. Efficient delivery is particularly vital for antisense phosphorodiamidate morpholino oligomers, which have a neutral backbone, and are known [...] Read more.
One of the crucial aspects of screening antisense oligonucleotides destined for therapeutic application is confidence that the antisense oligomer is delivered efficiently into cultured cells. Efficient delivery is particularly vital for antisense phosphorodiamidate morpholino oligomers, which have a neutral backbone, and are known to show poor gymnotic uptake. Here, we report several methods to deliver these oligomers into cultured cells. Although 4D-Nucleofector™ or Neon™ electroporation systems provide efficient delivery and use lower amounts of phosphorodiamidate morpholino oligomer, both systems are costly. We show that some readily available transfection reagents can be used to deliver phosphorodiamidate morpholino oligomers as efficiently as the electroporation systems. Among the transfection reagents tested, we recommend Lipofectamine 3000™ for delivering phosphorodiamidate morpholino oligomers into fibroblasts and Lipofectamine 3000™ or Lipofectamine 2000™ for myoblasts/myotubes. We also provide optimal programs for nucleofection into various cell lines using the P3 Primary Cell 4D-Nucleofector™ X Kit (Lonza), as well as antisense oligomers that redirect expression of ubiquitously expressed genes that may be used as positive treatments for human and murine cell transfections. Full article
(This article belongs to the Special Issue Antisense Oligonucleotide Chemistry and Applications)
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12 pages, 4423 KiB  
Article
Solid-Phase Synthesis of Phosphorothioate/Phosphonothioate and Phosphoramidate/Phosphonamidate Oligonucleotides
by Ondřej Kostov, Radek Liboska, Ondřej Páv, Pavel Novák and Ivan Rosenberg
Molecules 2019, 24(10), 1872; https://doi.org/10.3390/molecules24101872 - 15 May 2019
Cited by 4 | Viewed by 5515
Abstract
We have developed a robust solid-phase protocol which allowed the synthesis of chimeric oligonucleotides modified with phosphodiester and O-methylphosphonate linkages as well as their P-S and P-N variants. The novel O-methylphosphonate-derived modifications were obtained by oxidation, sulfurization, and amidation of the [...] Read more.
We have developed a robust solid-phase protocol which allowed the synthesis of chimeric oligonucleotides modified with phosphodiester and O-methylphosphonate linkages as well as their P-S and P-N variants. The novel O-methylphosphonate-derived modifications were obtained by oxidation, sulfurization, and amidation of the O-methyl-(H)-phosphinate internucleotide linkage introduced into the oligonucleotide chain by H-phosphonate chemistry using nucleoside-O-methyl-(H)-phosphinates as monomers. The H-phosphonate coupling followed by oxidation after each cycle enabled us to successfully combine H-phosphonate and phosphoramidite chemistries to synthesize diversely modified oligonucleotide strands. Full article
(This article belongs to the Special Issue Antisense Oligonucleotide Chemistry and Applications)
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15 pages, 7635 KiB  
Article
Systematic Screening of Commonly Used Commercial Transfection Reagents towards Efficient Transfection of Single-Stranded Oligonucleotides
by Tao Wang, Leon M. Larcher, Lixia Ma and Rakesh N. Veedu
Molecules 2018, 23(10), 2564; https://doi.org/10.3390/molecules23102564 - 08 Oct 2018
Cited by 114 | Viewed by 15092
Abstract
Non-viral vector-mediated transfection is a core technique for in vitro screening of oligonucleotides. Despite the growing interests in the development of oliogonucleotide-based drug molecules in recent years, a comprehensive comparison of the transfection efficacy of commonly used commercial transfection reagents has not been [...] Read more.
Non-viral vector-mediated transfection is a core technique for in vitro screening of oligonucleotides. Despite the growing interests in the development of oliogonucleotide-based drug molecules in recent years, a comprehensive comparison of the transfection efficacy of commonly used commercial transfection reagents has not been reported. In this study, five commonly used transfection reagents, including Lipofectamine 3000, Lipofectamine 2000, Fugene, RNAiMAX and Lipofectin, were comprehensively analyzed in ten cell lines using a fluorescence imaging-based transfection assay. Although the transfection efficacy and toxicity of transfection reagents varied depending on cell types, the toxicity of transfection reagents generally displayed a positive correlation with their transfection efficacy. According to our results, Lipofectamine 3000, Fugene and RNAiMAX showed high transfection efficacy, however, RNAiMAX may be a better option for majority of cells when lower toxicity is desired. The transfection efficacy of Lipofectamine 2000 was compromised by its high toxicity, which may adversely affect its application in most cells. We firmly believe that our findings may contribute to the future In vitro delivery and screening of single-stranded therapeutic oligonucleotides such as antisense oligonucleotides, antimiRs, and DNAzymes. Full article
(This article belongs to the Special Issue Antisense Oligonucleotide Chemistry and Applications)
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