Adeno-Associated Virus-Based Gene Therapy for Neurological Disorders
A special issue of Brain Sciences (ISSN 2076-3425). This special issue belongs to the section "Molecular and Cellular Neuroscience".
Deadline for manuscript submissions: 10 November 2024 | Viewed by 247
Special Issue Editors
Interests: neurological, neuromuscular and neurodegenerative diseases; gene therapy; AAV; in vitro modeling; lysosomal storage disorders
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Neurological disorders are among the most difficult pathologies to treat because of the limited access to brain structures, which are protected by physical barriers such as the blood–brain barrier (BBB); the complexity of the CNS; and the multitude of cell types involved in disease progression. Gene therapy is an alternative to traditional pharmacological approaches that has made important advances in the past few years for treating diseases of the nervous system. Adeno-associated viral vectors (AAVs) have opened the opportunity of widespread targeting of the CNS due to their non-integrative and low immunogenic profile. These vectors are now amongst the most commonly used for the purposes of either disease modeling or for the development of therapeutic strategies for neurological diseases. While exciting progress has been made, AAV gene therapy is still a young and emerging field that faces multiple challenges and hurdles such as manufacturing, safety concerns at high doses, delivery to deeper brain structures and more. This Special Issue aims to address the current challenges and potential solutions by showcasing the advances in the research of AAV gene therapy for all kind of neurological disorders. We welcome all papers of original research, critical reviews, clinical trials, etc.
Dr. Kathrin Meyer
Dr. Shibi Likhite
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 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. 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
- AAVs
- neurological disorders
- neurological disorders
- vitro model
