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Biomedicines
Special Issues
Antisense Therapy
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Antisense Therapy

A special issue of Biomedicines (ISSN 2227-9059). This special issue belongs to the section "Molecular and Translational Medicine".

Deadline for manuscript submissions: closed (30 November 2017) | Viewed by 43455

Special Issue Editor

Dr. Linda Popplewell

E-Mail Website
Guest Editor
Department of Biological Sciences, Royal Holloway University of London, Egham, UK
Interests: antisense oligonucleotides; exon skipping; muscular dystrophies; dystrophin; myostatin; muscle fibrosis; gene editing; gene addition therapy

Special Issue Information

Dear Colleagues,

This Special Issue, “Antisense Therapy”, will focus on the progress of antisense oligonucleotides as therapy for various conditions. Issues that need addressing are Duchenne muscular dystrophy (DMD) and exon skipping; spinal muscular dystrophy and antisense oligonucleotides; new chemistries; enhancing delivery; genetic modifiers; miRNAs and antagomirs.

We cordially invite authors in the field to submit review articles or original research to this Special Issue of Biomedicines.

Dr. Linda Popplewell
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. Biomedicines is an international peer-reviewed open access monthly 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 2600 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

  • DMD
  • SMA (Spinal muscular atrophy)
  • clinical trials
  • delivery
  • chemistries

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Published Papers (6 papers)

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Editorial

Jump to: Review

2 pages, 154 KiB  
Editorial
Editorial of the Special Issue: Antisense Therapies
by Linda J. Popplewell
Biomedicines 2018, 6(4), 95; https://doi.org/10.3390/biomedicines6040095 - 27 Sep 2018
Viewed by 2727
Abstract
n/a Full article
(This article belongs to the Special Issue Antisense Therapy)

Review

Jump to: Editorial

21 pages, 1269 KiB  
Review
Targeting TGFβ Signaling to Address Fibrosis Using Antisense Oligonucleotides
by James T. March, Golnoush Golshirazi, Viktorija Cernisova, Heidi Carr, Yee Leong, Ngoc Lu-Nguyen and Linda J. Popplewell
Biomedicines 2018, 6(3), 74; https://doi.org/10.3390/biomedicines6030074 - 25 Jun 2018
Cited by 20 | Viewed by 7680
Abstract
Fibrosis results from the excessive accumulation of extracellular matrix in chronically injured tissue. The fibrotic process is governed by crosstalk between many signaling pathways. The search for an effective treatment is further complicated by the fact that there is a degree of tissue-specificity [...] Read more.
Fibrosis results from the excessive accumulation of extracellular matrix in chronically injured tissue. The fibrotic process is governed by crosstalk between many signaling pathways. The search for an effective treatment is further complicated by the fact that there is a degree of tissue-specificity in the pathways involved, although the process is not completely understood for all tissues. A plethora of drugs have shown promise in pre-clinical models, which is not always borne out translationally in clinical trial. With the recent approvals of two antisense oligonucleotides for the treatment of the genetic diseases Duchenne muscular dystrophy and spinal muscular atrophy, we explore here the potential of antisense oligonucleotides to knockdown the expression of pro-fibrotic proteins. We give an overview of the generalized fibrotic process, concentrating on key players and highlight where antisense oligonucleotides have been used effectively in cellular and animal models of different fibrotic conditions. Consideration is given to the advantages antisense oligonucleotides would have as an anti-fibrotic therapy alongside factors that would need to be addressed to improve efficacy. A prospective outlook for the development of antisense oligonucleotides to target fibrosis is outlined. Full article
(This article belongs to the Special Issue Antisense Therapy)
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15 pages, 1048 KiB  
Review
Cell-Penetrating Peptides to Enhance Delivery of Oligonucleotide-Based Therapeutics
by Graham McClorey and Subhashis Banerjee
Biomedicines 2018, 6(2), 51; https://doi.org/10.3390/biomedicines6020051 - 5 May 2018
Cited by 127 | Viewed by 10184
Abstract
The promise of nucleic acid based oligonucleotides as effective genetic therapies has been held back by their low bioavailability and poor cellular uptake to target tissues upon systemic administration. One such strategy to improve upon delivery is the use of short cell-penetrating peptides [...] Read more.
The promise of nucleic acid based oligonucleotides as effective genetic therapies has been held back by their low bioavailability and poor cellular uptake to target tissues upon systemic administration. One such strategy to improve upon delivery is the use of short cell-penetrating peptides (CPPs) that can be either directly attached to their cargo through covalent linkages or through the formation of noncovalent nanoparticle complexes that can facilitate cellular uptake. In this review, we will highlight recent proof-of-principle studies that have utilized both of these strategies to improve nucleic acid delivery and discuss the prospects for translation of this approach for clinical application. Full article
(This article belongs to the Special Issue Antisense Therapy)
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11 pages, 260 KiB  
Review
RNA-Targeted Therapies and Amyotrophic Lateral Sclerosis
by Stéphane Mathis and Gwendal Le Masson
Biomedicines 2018, 6(1), 9; https://doi.org/10.3390/biomedicines6010009 - 15 Jan 2018
Cited by 20 | Viewed by 5957
Abstract
Amyotrophic lateral sclerosis (ALS) is a fatal motor disease in adults. Its pathophysiology remains mysterious, but tremendous advances have been made with the discovery of the most frequent mutations of its more common familial form linked to the C9ORF72 gene. Although most cases [...] Read more.
Amyotrophic lateral sclerosis (ALS) is a fatal motor disease in adults. Its pathophysiology remains mysterious, but tremendous advances have been made with the discovery of the most frequent mutations of its more common familial form linked to the C9ORF72 gene. Although most cases are still considered sporadic, these genetic mutations have revealed the role of RNA production, processing and transport in ALS, and may be important players in all ALS forms. There are no disease-modifying treatments for adult human neurodegenerative diseases, including ALS. As in spinal muscular atrophy, RNA-targeted therapies have been proposed as potential strategies for treating this neurodegenerative disorder. Successes achieved in various animal models of ALS have proven that RNA therapies are both safe and effective. With careful consideration of the applicability of such therapies in humans, it is possible to anticipate ongoing in vivo research and clinical trial development of RNA therapies for treating ALS. Full article
(This article belongs to the Special Issue Antisense Therapy)
12 pages, 953 KiB  
Review
Skipping Multiple Exons to Treat DMD—Promises and Challenges
by Tejal Aslesh, Rika Maruyama and Toshifumi Yokota
Biomedicines 2018, 6(1), 1; https://doi.org/10.3390/biomedicines6010001 - 2 Jan 2018
Cited by 41 | Viewed by 10217
Abstract
Duchenne muscular dystrophy (DMD) is a lethal disorder caused by mutations in the DMD gene. Antisense-mediated exon-skipping is a promising therapeutic strategy that makes use of synthetic nucleic acids to skip frame-disrupting exon(s) and allows for short but functional protein expression by restoring [...] Read more.
Duchenne muscular dystrophy (DMD) is a lethal disorder caused by mutations in the DMD gene. Antisense-mediated exon-skipping is a promising therapeutic strategy that makes use of synthetic nucleic acids to skip frame-disrupting exon(s) and allows for short but functional protein expression by restoring the reading frame. In 2016, the U.S. Food and Drug Administration (FDA) approved eteplirsen, which skips DMD exon 51 and is applicable to approximately 13% of DMD patients. Multiple exon skipping, which is theoretically applicable to 80–90% of DMD patients in total, have been demonstrated in animal models, including dystrophic mice and dogs, using cocktail antisense oligonucleotides (AOs). Although promising, current drug approval systems pose challenges for the use of a cocktail AO. For example, both exons 6 and 8 need to be skipped to restore the reading frame in dystrophic dogs. Therefore, the cocktail of AOs targeting these exons has a combined therapeutic effect and each AO does not have a therapeutic effect by itself. The current drug approval system is not designed to evaluate such circumstances, which are completely different from cocktail drug approaches in other fields. Significant changes are needed in the drug approval process to promote the cocktail AO approach. Full article
(This article belongs to the Special Issue Antisense Therapy)
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Graphical abstract

2558 KiB  
Review
The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders
by Philippine Aupy, Lucía Echevarría, Karima Relizani and Aurélie Goyenvalle
Biomedicines 2018, 6(1), 2; https://doi.org/10.3390/biomedicines6010002 - 22 Dec 2017
Cited by 12 | Viewed by 5886
Abstract
Antisense Oligonucleotides (ASOs) represent very attractive therapeutic compounds for the treatment of numerous diseases. The antisense field has remarkably progressed over the last few years with the approval of the first antisense drugs and with promising developments of more potent and nuclease resistant [...] Read more.
Antisense Oligonucleotides (ASOs) represent very attractive therapeutic compounds for the treatment of numerous diseases. The antisense field has remarkably progressed over the last few years with the approval of the first antisense drugs and with promising developments of more potent and nuclease resistant chemistries. Despite these recent clinical successes and advances in chemistry and design, effective delivery of ASOs to their target tissues remains a major issue. This review will describe the latest advances obtained with the tricyclo-DNA (tcDNA) chemistry which displays unique pharmacological properties and unprecedented uptake in many tissues after systemic administration. We will examine the variety of therapeutic approaches using both fully modified tcDNA-ASOs and gapmers, including splice switching applications, correction of aberrant splicing, steric blocking strategies and targeted gene knock-down mediated by RNase H recruitment. We will then discuss the merits and potential liabilities of the tcDNA chemistry in the context of ASO drug development. Full article
(This article belongs to the Special Issue Antisense Therapy)
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