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Special Issue "Gene Therapy for Neurodegenerative Disease"

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: 15 July 2021.

Special Issue Editors

Prof. Dr. Javier Díaz-Nido
E-Mail Website
Guest Editor
Departamento de Biología Molecular, Universidad Autónoma de Madrid, 28049 Madrid, Spain
Interests: Neurodegeneration; Neurodegenerative Diseases; Ataxias; Cerebellum; Neurotrophic Factors; Neuroprotection; Gene Therapy
Dr. S. Herranz-Martin
E-Mail Website
Guest Editor
Departamento de Biología Molecular, Universidad Autónoma de Madrid, 28049 Madrid, Spain
Interests: Gene therapy; cell therapy; AAV; Ataxias; mitochondria; Neurodegenerative diseases; neuroprotection
Prof. Dr. Ahad Rahim
E-Mail Website
Guest Editor
School of Pharmacy, University College London, London, UK
Interests: AAV; gene therapy; cell therapy; neurodegeneration; inherited metabolic diseases; lysosomal storage diseases

Special Issue Information

Dear Colleagues,

According to the Foof and Drug Administration (FDA) “gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use”. Within the past few decades, gene therapy has emerged as a very valuable tool to restore defective genes causing human disorders. This technology is especially attractive for the treatment of monogenic diseases. Many neurodegenerative conditions, especially rare disorders, are in fact, monogenic, making them, therefore, ideal for their treatment using gene therapy approaches. Different viral vectors, such as Adeno-associated virus (AAV), lentivirus (LV), retroviruses (RV), adenoviruses (Ad) or herpes simplex virus (HSV), among others, or non-viral vectors, have been widely used to carry the therapeutic gene into the host cell, showing promising results in hundreds of preclinical and clinical studies published. Indeed, some AAV-based gene therapy products, such as Zolgensma®, have been already approved by the FDA for the treatment of Spinal Muscular Atrophy (SMA), a fatal neurodegenerative disease affecting children. However, there still remain many important challenges for the development of effective gene therapies for neurological disorders. In a year in which pandemic has made that many laboratories have focused their efforts in the fight against SARS-CoV-2, it is very important to also advance in the research of many other disorders including those affecting the central nervous system. Thus, the aim of this special issue will be to cover preclinical studies and basic science studies using gene therapy approaches for the treatment of different neurodegenerative diseases.

Prof. Dr. Javier Díaz-Nido
Dr. S. Herranz-Martin
Prof. Dr. Ahad Rahim
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. 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

  • Gene therapy
  • Neurodegeneration
  • Neuroprotection
  • Neurotoxicity
  • Viral vector
  • AAV
  • Transduction
  • Non-viral vector

Published Papers (1 paper)

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Review

Open AccessReview
Future Prospects of Gene Therapy for Friedreich’s Ataxia
Int. J. Mol. Sci. 2021, 22(4), 1815; https://0-doi-org.brum.beds.ac.uk/10.3390/ijms22041815 - 11 Feb 2021
Viewed by 917
Abstract
Friedreich’s ataxia is an autosomal recessive neurogenetic disease that is mainly associated with atrophy of the spinal cord and progressive neurodegeneration in the cerebellum. The disease is caused by a GAA-expansion in the first intron of the frataxin gene leading to a decreased [...] Read more.
Friedreich’s ataxia is an autosomal recessive neurogenetic disease that is mainly associated with atrophy of the spinal cord and progressive neurodegeneration in the cerebellum. The disease is caused by a GAA-expansion in the first intron of the frataxin gene leading to a decreased level of frataxin protein, which results in mitochondrial dysfunction. Currently, there is no effective treatment to delay neurodegeneration in Friedreich’s ataxia. A plausible therapeutic approach is gene therapy. Indeed, Friedreich’s ataxia mouse models have been treated with viral vectors en-coding for either FXN or neurotrophins, such as brain-derived neurotrophic factor showing promising results. Thus, gene therapy is increasingly consolidating as one of the most promising therapies. However, several hurdles have to be overcome, including immunotoxicity and pheno-toxicity. We review the state of the art of gene therapy in Friedreich’s ataxia, addressing the main challenges and the most feasible solutions for them. Full article
(This article belongs to the Special Issue Gene Therapy for Neurodegenerative Disease)
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