Gene Therapy for Neurological Diseases
A special issue of Brain Sciences (ISSN 2076-3425). This special issue belongs to the section "Molecular and Cellular Neuroscience".
Deadline for manuscript submissions: 30 October 2026 | Viewed by 207
Special Issue Editors
2. Department of Biochemistry and Molecular Biology, School of Veterinary Medicine, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain
3. Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain
Interests: gene therapy; adeno-associated viral vectors; lysosomal storage diseases; mucopolysaccharidosis; neurological diseases; bone diseases; immune responses to AAV; diabetes and obesity
2. Department of Biochemistry and Molecular Biology, School of Veterinary Medicine, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain
3. Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain
Interests: gene therapy; adeno-associated viral vectors; lysosomal storage diseases; diabetes and obesity
2. Department of Immunology, Harvard Medical School, Boston, MA 02115, USA
3. Harvard Department of Stem Cell and Regenerative Biology, Cambridge, MA 02138, USA
Interests: gene therapy; cell therapy; neurodegenerative diseases; diabetes; cancer; immunology
Special Issue Information
Dear Colleagues,
Gene therapy has emerged as a promising approach for treating a broad spectrum of neurological diseases, from monogenic disorders to complex neurodegenerative and neurodevelopmental conditions.
Recent progress in vector engineering has expanded the range of strategies available to treat or modulate disease-relevant pathways in the central nervous system (CNS). Despite this potential, delivering therapeutic vectors efficiently and safely to the CNS remains a major challenge, largely due to the restrictive nature of the blood–brain barrier, the limited penetration after systemic administration, and the risk of local and systemic immune responses.
This Special Issue, titled“Gene Therapy for Neurological Diseases, aims to showcase innovative preclinical and clinical research addressing these opportunities and challenges in the CNS. It will focus on novel vector platforms and delivery routes, strategies to overcome BBB, and new therapeutic approaches to enhance safety and long-term efficacy and durability of gene therapy approaches.
We invite submissions that address mechanistic studies, innovative therapeutic designs, immune responses, as well as translational and early clinical studies across diverse neurological indications. Original research articles, reviews, and brief communications are welcome to foster interdisciplinary collaboration and accelerate the development of next-generation gene therapies for patients with neurological disorders.
Dr. Sara Marcó
Dr. Miquel García
Dr. Xavier Sanchez
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 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 250 words) can be sent to the Editorial Office for assessment.
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. Brain Sciences 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 2200 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
- gene therapy
- neurological diseases
- adeno-associated viral vectors
- lentiviral vectors
- lipid nanoparticles
- gene replacement
- genome editing
- lipid nanoparticles
- immune responses
- translational neuroscience
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