Special Issue "Neuromuscular Disorders: Current Gene and Cell Therapeutic Approaches"

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Biopharmaceuticals".

Deadline for manuscript submissions: 30 September 2021.

Special Issue Editors

Prof. Dr. Leonidas A. Phylactou
E-Mail Website
Guest Editor
Dr. Andrie Koutsoulidou
E-Mail Website
Guest Editor
Department of Molecular Genetics, Cyprus Institute of Neurology & Genetics, Nicosia, Cyprus
Interests: muscular dystrophies; gene therapy; biomarkers; miRNAs

Special Issue Information

Dear Colleagues,

Neuromuscular disorders are a heterogenic group of diseases affecting the muscular system or the peripheral nervous system. Some of these conditions are also associated with the central nervous system, such as amyotrophic lateral sclerosis (ALS). Neuromuscular disorders are predominantly characterized by progressive muscle weakness. Currently, no therapeutic treatments have been identified for any of these disorders. The high heterogeneity with respect to clinical severity, the cause of the disease, and the primary cell type that is affected (e.g., motor neurons, skeletal muscle, and Schwann cells) are considered the main challenges for the development of a successful therapeutic strategy for this group of disorders. However, in recent years, research into therapy development for neuromuscular disorders has made remarkable progress. Innovative therapeutic approaches have been applied to specific neuromuscular diseases with encouraging results.

This Special Issue will cover the recent progress in developing therapeutic and diagnostic strategies for neuromuscular disorders through a series of original research articles and reviews from experts in the field. Therapeutic approaches involving the use of nucleic acid oligonucleotides such as antisense oligonucleotides and miRNAs, biopharmaceutical compounds, genome editing, and different delivery tools including viral vectors and nanoparticles will be highlighted in this Special Issue.

Prof. Dr. Leonidas A. Phylactou
Dr. Andrie Koutsoulidou
Guest Editors

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 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. Pharmaceuticals 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 1800 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

  • Muscular dystrophies
  • Motor neuron diseases
  • Myopathies
  • Cell-based therapy
  • Gene therapy
  • Antisense oligonucleotides
  • miRNAs
  • Pharmacological strategies
  • Stem cells
  • Viral vectors
  • Nanoparticles

Published Papers (1 paper)

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Review

Review
Stem Cell Models and Gene Targeting for Human Motor Neuron Diseases
Pharmaceuticals 2021, 14(6), 565; https://0-doi-org.brum.beds.ac.uk/10.3390/ph14060565 - 12 Jun 2021
Viewed by 853
Abstract
Motor neurons are large projection neurons classified into upper and lower motor neurons responsible for controlling the movement of muscles. Degeneration of motor neurons results in progressive muscle weakness, which underlies several debilitating neurological disorders including amyotrophic lateral sclerosis (ALS), hereditary spastic paraplegias [...] Read more.
Motor neurons are large projection neurons classified into upper and lower motor neurons responsible for controlling the movement of muscles. Degeneration of motor neurons results in progressive muscle weakness, which underlies several debilitating neurological disorders including amyotrophic lateral sclerosis (ALS), hereditary spastic paraplegias (HSP), and spinal muscular atrophy (SMA). With the development of induced pluripotent stem cell (iPSC) technology, human iPSCs can be derived from patients and further differentiated into motor neurons. Motor neuron disease models can also be generated by genetically modifying human pluripotent stem cells. The efficiency of gene targeting in human cells had been very low, but is greatly improved with recent gene editing technologies such as zinc-finger nucleases (ZFN), transcription activator-like effector nucleases (TALEN), and CRISPR-Cas9. The combination of human stem cell-based models and gene editing tools provides unique paradigms to dissect pathogenic mechanisms and to explore therapeutics for these devastating diseases. Owing to the critical role of several genes in the etiology of motor neuron diseases, targeted gene therapies have been developed, including antisense oligonucleotides, viral-based gene delivery, and in situ gene editing. This review summarizes recent advancements in these areas and discusses future challenges toward the development of transformative medicines for motor neuron diseases. Full article
(This article belongs to the Special Issue Neuromuscular Disorders: Current Gene and Cell Therapeutic Approaches)
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