Gene Therapy: Where We Are and Where to Go?
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 (29 February 2024) | Viewed by 509
Special Issue Editor
Interests: neuromuscular disorders; congenital myopathies; muscular dystrophies; preclinical trials; gene therapy; transgenic animal models
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
This Special Issue, titled “Gene Therapy: Where We Are and Where to Go”, will aim at giving an up-to-date and comprehensive overview of recent advances in gene therapy therapeutic strategies. We invite researchers in the field to submit original research and review articles focused on the latest progress in preclinical and clinical approaches in ex vivo and in vivo gene replacement, gene silencing, gene repair and other genome-editing methods, notably base-editing, which enables precise modifications of the human genome sequences, using genetic, pharmacological or oligonucleotide-based molecules. The scope of this Special Issue encompasses delivery and targeting strategies such as viral vector approaches (notably through Adeno-Associated Virus (AAV) and Lentiviral vectors), CAR-T cell therapy, non-viral drug-delivery particles and any other original strategies. Topics may include (but are not limited to) results of efficacy and toxicology studies on in silico, cellular and animal disease models; new vector development; results of clinical trials; efficacy and toxicity biomarker discovery and validation; and functional genomics for upheaving our understanding of human genomic regulation. Studies performed on genetic disorders, cancer and any other relevant pathologies will be considered based on scientific novelty and general interest. Taking into consideration the recent drawbacks due to immunological reactions to viral vectors and the ever-pending question of treatment re-administration/administration in seropositive patients, articles related to methods for temporarily clearing antibodies from circulation or evading detection by the host immune system are especially welcomed. Methodologies such as nanoparticle-mediated deliveries, development of new immune-blind capsids, use of “immune stealth” allogeneic universal cells, development of IgG-cleaving molecules and innovative patient-immunomonitoring tests would be of great interest.
Dr. Nathalie Daniele
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
- gene therapy
- Adeno-Associated Virus
- Lentiviral vectors
- gene replacement
- gene editing
- oligonucleotide-based therapy
- pharmacologically based gene therapy
- toxicity
- biomarkers
- immune response
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.
- Reprint: MDPI Books provides the opportunity to republish successful Special Issues in book format, both online and in print.
Further information on MDPI's Special Issue policies can be found here.
