ijms-logo

Journal Browser

Journal Browser

Understanding and Treating Amyotrophic Lateral Sclerosis and Related Motor Neuron Diseases

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Neurobiology".

Deadline for manuscript submissions: 20 May 2025 | Viewed by 470

Special Issue Editor


E-Mail Website
Guest Editor
1. Imaging Brain & Neuropsychiatry iBraiN U1253, Institut National de la Santé et de la Recherche Médicale (INSERM), Université de Tours, 37032 Tours, France
2. Service de Biochimie et Biologie Moléculaire, CHU de Tours, 37044 Tours, France
Interests: ALS; genetics; neurobiology; post-translational modifications; aggregation; neurogenesis

Special Issue Information

Dear Colleagues,

Our understanding of the pathophysiology of amyotrophic lateral sclerosis (ALS) and its related motor neuron diseases has progressed dramatically in recent years, leading to some very interesting reflections on their definition and heterogeneity, as well as to numerous preclinical and clinical studies on potential therapeutic approaches. Some ALS/MND clinicians have indicated that this is a time for optimism. However, there are still enormous challenges to overcome. We need to better understand the complexity of these diseases, i.e., describe the molecular mechanisms involved at different stages; identify overlaps between these pathologies or with other pathologies; search for new, robust biomarkers and link them to clinical heterogeneity, disease progression, and response to therapeutic approaches; intensify research on the causes of ALS/MND, which are still unknown in the majority of patients; determine new mutations, genetic factors (causal, risk, progression), and epigenetics factors, etc.; better study the roles played by the various cell types in the motor neuron environment; and better characterize existing and new in vitro and in vivo study models. This Special Issue invites manuscript submissions that address these and any other questions that will help to better understand ALS/MND at the molecular level.

Prof. Dr. Patrick Vourc'h
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. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. There is an Article Processing Charge (APC) for publication in this open access journal. For details about the APC please see here. 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

  • neurodegenerative
  • ALS
  • motor neuron
  • genetics
  • molecular pathways
  • biomarkers
  • models
  • treatments

Benefits of Publishing in a Special Issue

  • Ease of navigation: Grouping papers by topic helps scholars navigate broad scope journals more efficiently.
  • Greater discoverability: Special Issues support the reach and impact of scientific research. Articles in Special Issues are more discoverable and cited more frequently.
  • Expansion of research network: Special Issues facilitate connections among authors, fostering scientific collaborations.
  • External promotion: Articles in Special Issues are often promoted through the journal's social media, increasing their visibility.
  • e-Book format: Special Issues with more than 10 articles can be published as dedicated e-books, ensuring wide and rapid dissemination.

Further information on MDPI's Special Issue policies can be found here.

Published Papers (1 paper)

Order results
Result details
Select all
Export citation of selected articles as:

Review

21 pages, 1670 KiB  
Review
Targets and Gene Therapy of ALS (Part 1)
by Olga Shiryaeva, Christina Tolochko, Tatiana Alekseeva and Vyacheslav Dyachuk
Int. J. Mol. Sci. 2025, 26(9), 4063; https://doi.org/10.3390/ijms26094063 - 25 Apr 2025
Viewed by 311
Abstract
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the selective death of motor neurons, which causes muscle atrophy. Genetic forms of ALS are recorded only in 10% of cases. However, over the past decade, studies in genetics have substantially contributed to [...] Read more.
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the selective death of motor neurons, which causes muscle atrophy. Genetic forms of ALS are recorded only in 10% of cases. However, over the past decade, studies in genetics have substantially contributed to our understanding of the molecular mechanisms underlying ALS. The identification of key mutations such as SOD1, C9orf72, FUS, and TARDBP has led to the development of targeted therapy that is gradually being introduced into clinical trials, opening up a broad range of opportunities for correcting these mutations. In this review, we aimed to present an extensive overview of the currently known mechanisms of motor neuron degeneration associated with mutations in these genes and also the gene therapy methods for inhibiting the expression of their mutant proteins. Among these, antisense oligonucleotides, RNA interference (siRNA and miRNA), and gene-editing (CRISPR/Cas9) methods are of particular interest. Each has shown its efficacy in animal models when targeting mutant genes, whereas some of them have proven to be efficient in human clinical trials. Full article
Show Figures

Figure 1

Back to TopTop