Gene Transfer in Brain Disorders
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: 30 November 2024 | Viewed by 4470
Special Issue Editors
Interests: gene transfer/therapy; transgenic mice; hippocampus rejuvenation; brain disorders; intellectual ability and disability
Special Issues, Collections and Topics in MDPI journals
Interests: gene therapy; AAV vectors; Alzheimer's disease; Parkinson's disease; Niemann-Pick disease
Interests: psychiatry; behavioral health; dentate granular neurons; mindfulness based intervention; intensive care unit; depression; social media; mood; anxiety; stress
Special Issue Information
Dear Colleagues,
Of all the organs in the body, the brain—protected by the endothelial blood–brain barrier (BBB), which regulates the internal milieu—is the most complicated in terms of the numbers of structures/functions involved, and the numbers of gene transcripts that change over time. The pathogenesis of the brain can be viewed via the lens of the dysregulation of gene variants expressions that are reported, not only in intellectual disabilities (i.e., Down syndrome, Rett syndrome, DiGeorge syndrome, Fragile X syndrome, autism spectrum disorders), and age-regulated neurodegenerative diseases, (i.e., Alzheimer, Huntington, Parkinson’s diseases), but also in the etiology of epilepsy, brain trauma, brain cancer, Creutzfeldt–Jakob diseases, and other complex psychiatric diseases (schizophrenia, bipolar disorder, substance use disorders). Partially due to the BBB, most of the pharmacological treatments have limited efficacy, whereas surgical or neuromodulation interventions have limited indications. In contrast, gene transfer may specifically target and correct the underlying mechanisms of a disease. Various viral vector types (including retrovirus vectors, lentivirus vectors, adenovirus vectors, herpes simplex virus type 1) have been recently used for phase I/II clinical trials. Adeno-associated virus (AAV) vectors, with their well-defined in vivo safety profile, have emerged as one of the most important vectors for the treatment of brain disorders, including the delivery of CRISPR-Cas9 genome-editing machinery.
Dr. Tam Thanh Quach
Dr. Lluís Samaranch
Dr. Anne-Marie Duchemin
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. 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
- intellectual disability
- neurodegenerative disorders
- psychiatric diseases
- gene transfer
- AAV
Benefits of Publishing in a Special Issue
- Ease of navigation: Grouping papers by topic helps scholars navigate broad scope journals more efficiently.
- Greater discoverability: Special Issues support the reach and impact of scientific research. Articles in Special Issues are more discoverable and cited more frequently.
- Expansion of research network: Special Issues facilitate connections among authors, fostering scientific collaborations.
- External promotion: Articles in Special Issues are often promoted through the journal's social media, increasing their visibility.
- e-Book format: Special Issues with more than 10 articles can be published as dedicated e-books, ensuring wide and rapid dissemination.
Further information on MDPI's Special Issue polices can be found here.
