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Disease Mechanisms and Therapeutics for ALS: From Animal Models to...
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Disease Mechanisms and Therapeutics for ALS: From Animal Models to Humans

A special issue of Life (ISSN 2075-1729). This special issue belongs to the section "Pharmaceutical Science".

Deadline for manuscript submissions: closed (31 July 2020) | Viewed by 4805

Special Issue Editor

Dr. Eiichi Tokuda

E-Mail Website
Guest Editor
School of Pharmacy, Nihon University, 7-7-1, Narashinodai, Funabashi, Chiba 274-8555, Japan
Interests: amyotrophic lateral sclerosis; protein aggregation; endocytosis; metal homeostasis

Special Issue Information

Dear Colleagues,

Amyotrophic lateral sclerosis (ALS) is an incurable neuromuscular disease characterized by the selective death of motor neurons, resulting in progressive muscle atrophy. There is an emergent need to develop therapeutic strategies because of the modest effects of Riluzole and Edaravone, approved drugs for use in ALS, on the disease process. Success of developing animal models based on genetic discovery provides an understanding of molecular mechanisms underlying the pathogenesis of ALS as well as an opportunity to identify a potential candidate for treatment. Much effort has been devoted to discovering responsible genes for ALS such as SOD1, TARDBP, FUS, and C9ORF72. Nevertheless, our understanding of the disease process and the development of therapeutics for ALS can be still space for improvement.

This Special Issue welcomes original research with focus on a proof-concept study for a disease mechanism of ALS as well as a preclinical test using either mammalian or non-mammalian models carrying any responsible genes for ALS. Review articles that summarizes the ALS pathogenesis and/or preclinical outcomes based on animal models of ALS are also welcome. This Special Issue will advance our understanding of disease pathogenesis and develop therapeutic strategies for ALS with your research.

Dr. Eiichi Tokuda
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. Life 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

  • amyotrophic lateral sclerosis
  • animal models
  • preclinical study
  • disease mechanism
  • therapeutic strategy

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Published Papers (2 papers)

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Review

16 pages, 1230 KiB  
Review
Expression Changes of miRNAs in Humans and Animal Models of Amyotrophic Lateral Sclerosis and Their Potential Application for Clinical Diagnosis
by Ruili Wang, Liang Chen, Yuning Zhang, Bo Sun and Mengyao Liang
Life 2024, 14(9), 1125; https://doi.org/10.3390/life14091125 - 6 Sep 2024
Viewed by 823
Abstract
Amyotrophic lateral sclerosis (ALS) is a severe motor neuron disease. Current detection methods can only confirm the diagnosis at the onset of the disease, missing the critical window for early treatment. Recent studies using animal models have found that detecting changes in miRNA [...] Read more.
Amyotrophic lateral sclerosis (ALS) is a severe motor neuron disease. Current detection methods can only confirm the diagnosis at the onset of the disease, missing the critical window for early treatment. Recent studies using animal models have found that detecting changes in miRNA sites can predict the onset and severity of the disease in its early stages, facilitating early diagnosis and treatment. miRNAs show expression changes in motor neurons that connect the brain, spinal cord, and brain stem, as well as in the skeletal muscle in mouse models of ALS. Clinically, expression changes in some miRNAs in patients align with those in mouse models, such as the upregulation of miR-29b in the brain and the upregulation of miR-206 in the skeletal muscle. This study provides an overview of some miRNA study findings in humans as well as in animal models, including SOD1, FUS, TDP-43, and C9orf72 transgenic mice and wobbler mice, highlighting the potential of miRNAs as diagnostic markers for ALS. miR-21 and miR-206 are aberrantly expressed in both mouse model and patient samples, positioning them as key potential diagnostic markers in ALS. Additionally, miR-29a, miR-29b, miR-181a, and miR-142-3p have shown aberrant expression in both types of samples and show promise as clinical targets for ALS. Finally, miR-1197 and miR-486b-5p have been recently identified as aberrantly expressed miRNAs in mouse models for ALS, although further studies are needed to determine their viability as diagnostic targets. Full article
(This article belongs to the Special Issue Disease Mechanisms and Therapeutics for ALS: From Animal Models to Humans)
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14 pages, 245 KiB  
Review
Copper-ATSM as a Treatment for ALS: Support from Mutant SOD1 Models and Beyond
by Sara Nikseresht, James B.W. Hilton, Kai Kysenius, Jeffrey R. Liddell and Peter J. Crouch
Life 2020, 10(11), 271; https://doi.org/10.3390/life10110271 - 4 Nov 2020
Cited by 23 | Viewed by 3532
Abstract
The blood–brain barrier permeant, copper-containing compound, CuII(atsm), has successfully progressed from fundamental research outcomes in the laboratory through to phase 2/3 clinical assessment in patients with the highly aggressive and fatal neurodegenerative condition of amyotrophic lateral sclerosis (ALS). The most compelling [...] Read more.
The blood–brain barrier permeant, copper-containing compound, CuII(atsm), has successfully progressed from fundamental research outcomes in the laboratory through to phase 2/3 clinical assessment in patients with the highly aggressive and fatal neurodegenerative condition of amyotrophic lateral sclerosis (ALS). The most compelling outcomes to date to indicate potential for disease-modification have come from pre-clinical studies utilising mouse models that involve transgenic expression of mutated superoxide dismutase 1 (SOD1). Mutant SOD1 mice provide a very robust mammalian model of ALS with high validity, but mutations in SOD1 account for only a small percentage of ALS cases in the clinic, with the preponderant amount of cases being sporadic and of unknown aetiology. As per other putative drugs for ALS developed and tested primarily in mutant SOD1 mice, this raises important questions about the pertinence of CuII(atsm) to broader clinical translation. This review highlights some of the challenges associated with the clinical translation of new treatment options for ALS. It then provides a brief account of pre-clinical outcomes for CuII(atsm) in SOD1 mouse models of ALS, followed by an outline of additional studies which report positive outcomes for CuII(atsm) when assessed in cell and mouse models of neurodegeneration which do not involve mutant SOD1. Clinical evidence for CuII(atsm) selectively targeting affected regions of the CNS in patients is also presented. Overall, this review summarises the existing evidence which indicates why clinical relevance of CuII(atsm) likely extends beyond the context of cases of ALS caused by mutant SOD1. Full article
(This article belongs to the Special Issue Disease Mechanisms and Therapeutics for ALS: From Animal Models to Humans)
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