Special Issue "Mechanisms of and Solutions for Diseases Caused by Toxic RNA"

A special issue of Toxins (ISSN 2072-6651).

Deadline for manuscript submissions: 31 October 2021.

Special Issue Editor

Prof. Dr. Sybille Krauß
E-Mail Website
Guest Editor
University of Siegen, Faculty IV: School of Science and Technology, Institute of Biology / Human Biology, Adolf-Reichwein-Str. 2, 57076 Siegen, Germany
Interests: RNA–protein interaction; neurodegeneration; CAG repeat expansion; translation initiation; splicing

Special Issue Information

Dear Colleagues,

RNA is an important molecule that plays an essential role in both healthy tissue and disease development. Besides coding for proteins, RNA molecules have a diverse range of regulatory functions, including non-coding RNAs that regulate gene expression via RNA interference, ribosomal RNAs, tRNAs, and RNAs that regulate splicing. An RNA’s function depends on the correct three-dimensional structure of the RNA molecule, since interaction partners, such as RNA-binding proteins, recognize specific structural motifs.

Abnormal folding of an RNA, for example due to genetic mutation, can be associated with disease development. For example, expansion mutations of repetitive sequences, such as CAG repeats, lead to an aberrant hairpin structure in the mutant RNA molecule that is not present in the normal RNA. This aberrant hairpin structure can then recruit RNA-binding proteins, which can lead to both the mutant RNA and the RNA-binding proteins having abnormal activity as well as a loss of physiological function of the RNA-binding proteins. In this way, RNA can deregulate a diverse range of cellular pathways and, thus, be toxic. However, the question remains: what are the precise molecular mechanisms underlying RNA-mediated toxicity? Additionally, how can we target toxic RNA species in therapeutic approaches?

These questions are currently under investigation.

The focus of this Special Issue of Toxins will be on molecular mechanisms underlying diseases that are caused by toxic RNA as well as approaches to combating RNA-mediated toxicity.

Prof. Dr. Sybille Krauß
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 papers will be 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 double-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Toxins 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 2400 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

  • RNA
  • three-dimensional structure
  • RNA-mediated toxicity
  • RNA–protein interaction
  • RNA-targeting compounds
  • RNA silencing

Published Papers (1 paper)

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Review

Review
Huntingtin and Its Role in Mechanisms of RNA-Mediated Toxicity
Toxins 2021, 13(7), 487; https://doi.org/10.3390/toxins13070487 - 14 Jul 2021
Viewed by 711
Abstract
Huntington’s disease (HD) is caused by a CAG-repeat expansion mutation in the Huntingtin (HTT) gene. It is characterized by progressive psychiatric and neurological symptoms in combination with a progressive movement disorder. Despite the ubiquitous expression of HTT, pathological changes occur quite selectively in [...] Read more.
Huntington’s disease (HD) is caused by a CAG-repeat expansion mutation in the Huntingtin (HTT) gene. It is characterized by progressive psychiatric and neurological symptoms in combination with a progressive movement disorder. Despite the ubiquitous expression of HTT, pathological changes occur quite selectively in the central nervous system. Since the discovery of HD more than 150 years ago, a lot of research on molecular mechanisms contributing to neurotoxicity has remained the focal point. While traditionally, the protein encoded by the HTT gene remained the cynosure for researchers and was extensively reviewed elsewhere, several studies in the last few years clearly indicated the contribution of the mutant RNA transcript to cellular dysfunction as well. In this review, we outline recent studies on RNA-mediated molecular mechanisms that are linked to cellular dysfunction in HD models. These mechanisms include mis-splicing, aberrant translation, deregulation of the miRNA machinery, deregulated RNA transport and abnormal regulation of mitochondrial RNA. Furthermore, we summarize recent therapeutical approaches targeting the mutant HTT transcript. While currently available treatments are of a palliative nature only and do not halt the disease progression, recent clinical studies provide hope that these novel RNA-targeting strategies will lead to better therapeutic approaches. Full article
(This article belongs to the Special Issue Mechanisms of and Solutions for Diseases Caused by Toxic RNA)
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