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Gene Therapy for Neurological Disorder
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Gene Therapy for Neurological Disorder

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

Deadline for manuscript submissions: closed (28 February 2022) | Viewed by 7605

Special Issue Editor

Prof. Dr. Enrico Maria Surace

E-Mail Website
Guest Editor
Department of Translational Medicine, University of Naples Federico II, 80131 Naples, Italy
Interests: retina; gene therapy; adeno-associated virus (AAV) vector; transcription; transcriptional repression; zinc finger; transcription factors

Special Issue Information

Dear Colleagues,

Neurological disorders cause a higher burden than any other group of diseases urging the development of therapeutic interventions. Unprecedently, these diseases are benefiting of recent progress of genetic therapies. RNA interference, Antisense Oligonucleotides (ASO), and DNA-binding platforms for gene-editing and transcriptional regulation are enabling to intervene at genome, transcriptional, and transcript levels. Furthermore, large-scale genomic studies are currently multiplying the identification of the disease targets underlying monogenic and multifactorial neurological disorders. Finally, driven by the continuous development of the delivery systems, including engineered capsids and the delivery administration techniques, genetic therapies for neurological disorders are gaining more and more momentum.

In this regard, we invite investigators to contribute original research articles and review articles that will expand the way to tackle monogenic and multifactorial neurological disorders affecting the brain, the peripheral nervous system, including those causing chronic pain, and the sense organs such as the retina and the inner ear via genetic therapies approaches.

Prof. Dr. Enrico Maria Surace
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

Neurological disorders; Genetic therapies; Neuropathic pain; Cognition; Macular degeneration age-related; Neurometabolic diseases; Neuromuscular diseases; Retinal degeneration; Inner ear; Alzheimer's disease (AD); Angelman’s syndrome (AS); Attention deficit-hyperactivity disorder (ADHD); Autism spectrum disorder (ASD); Cornelia de Lange syndrome (CDLS); Dravet syndrome (DRVT); fragile X syndrome; Friedreich ataxia (FA); Huntington's disease (HD); Parkinson's disease (PD); Rett’s syndrome (RS); Spinal muscular atrophy (SMA); Spinocerebellar ataxia (SCA); Tuberous sclerosis (TS)

 

Published Papers (2 papers)

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Research

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15 pages, 3277 KiB  
Article
Schwannoma Gene Therapy via Adeno-Associated Viral Vector Delivery of Apoptosis-Associated Speck-like Protein Containing CARD (ASC): Preclinical Efficacy and Safety
by Sherif G. Ahmed, Casey A. Maguire, Shiliang Alice Cao and Gary J. Brenner
Int. J. Mol. Sci. 2022, 23(2), 819; https://0-doi-org.brum.beds.ac.uk/10.3390/ijms23020819 - 13 Jan 2022
Cited by 2 | Viewed by 2024
Abstract
Schwannomas are tumors derived from Schwann-lineage cells, cells that protect and support myelinated nerves in the peripheral nervous system. They are typically slow-growing, encapsulated and benign. These tumors develop along peripheral, spinal and cranial nerves causing pain, sensory-motor dysfunction and death. Primary treatment [...] Read more.
Schwannomas are tumors derived from Schwann-lineage cells, cells that protect and support myelinated nerves in the peripheral nervous system. They are typically slow-growing, encapsulated and benign. These tumors develop along peripheral, spinal and cranial nerves causing pain, sensory-motor dysfunction and death. Primary treatment for schwannoma is operative resection which can be associated with significant morbidity. Pharmacotherapy is largely restricted to bevacizumab, which has minimal or no efficacy for many patients and can be associated with treatment-limiting adverse effects. Given the suffering and morbidity associated with schwannoma and the paucity of therapeutic options, there is an urgent need for safe and effective therapies for schwannomas. We previously demonstrated that adeno-associated virus serotype 1 (AAV1) vector mediated delivery of the inflammasome adaptor protein, apoptosis-associated speck-like protein containing a caspase recruitment domain (ASC) under the control of the P0 promoter, produced a prolonged reduction in tumor volume and tumor-associated pain in human xenograft and mouse syngeneic schwannoma models. Here, we present data essential for the translation of our AAV1-P0-ASC schwannoma gene therapy to clinical trials. We determine the minimum effective dose of AAV1-P0-hASC required to induce an anti-tumor effect in the xenograft human-schwannoma model. We also show that the presence of preexisting AAV1 immunity does not alter the antitumor efficacy of AAV-P0-mASC in a syngeneic mouse schwannoma model. Furthermore, the maximum deliverable intratumoral dose of AAV1-P0-ASC was not associated with neuronal toxicity in immunocompetent mice. Taken together, these safety and efficacy data support the translation of the AAV1-P0-ASC schwannoma gene therapy strategy to clinical trials. Full article
(This article belongs to the Special Issue Gene Therapy for Neurological Disorder)
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Review

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12 pages, 672 KiB  
Review
Induced Pluripotent Stem Cells (iPSCs) and Gene Therapy: A New Era for the Treatment of Neurological Diseases
by Giulia Paolini Sguazzi, Valentina Muto, Marco Tartaglia, Enrico Bertini and Claudia Compagnucci
Int. J. Mol. Sci. 2021, 22(24), 13674; https://0-doi-org.brum.beds.ac.uk/10.3390/ijms222413674 - 20 Dec 2021
Cited by 11 | Viewed by 4971
Abstract
To date, gene therapy has employed viral vectors to deliver therapeutic genes. However, recent progress in molecular and cell biology has revolutionized the field of stem cells and gene therapy. A few years ago, clinical trials started using stem cell replacement therapy, and [...] Read more.
To date, gene therapy has employed viral vectors to deliver therapeutic genes. However, recent progress in molecular and cell biology has revolutionized the field of stem cells and gene therapy. A few years ago, clinical trials started using stem cell replacement therapy, and the induced pluripotent stem cells (iPSCs) technology combined with CRISPR-Cas9 gene editing has launched a new era in gene therapy for the treatment of neurological disorders. Here, we summarize the latest findings in this research field and discuss their clinical applications, emphasizing the relevance of recent studies in the development of innovative stem cell and gene editing therapeutic approaches. Even though tumorigenicity and immunogenicity are existing hurdles, we report how recent progress has tackled them, making engineered stem cell transplantation therapy a realistic option. Full article
(This article belongs to the Special Issue Gene Therapy for Neurological Disorder)
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