Special Issue "Gene Therapy"
Deadline for manuscript submissions: closed (31 August 2018).
Prof. Dr. Bart De Geest
Center for Molecular and Vascular Biology, Department of Cardiovascular Sciences, Katholieke Universiteit Leuven, Campus Gasthuisberg, Herestraat 49 bus 911, 3000 Leuven, Belgium
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Interests: hepatocyte-directed gene therapy; adeno-associated viral gene therapy; adenoviral gene therapy; non-viral gene therapy; familial hypercholesterolemia; low-density lipoprotein (LDL) receptor; high-density lipoproteins (HDL); heart failure; HDL-targeted therapies
Recombinant DNA technology provides a platform for the creation of functional gene-expressing units. The effective and safe delivery of these therapeutic agents in vivo is a prerequisite for successful gene therapy and has been a major challenge for gene therapists. Gene delivery vehicles or vectors are categorized into non-viral vectors and viral vectors. Non-viral vectors possess several advantages over viral vectors, such as easy scale-up production, the potential to carry large genes, and the absence of viral components with consequent low immunotoxicity. However, non-viral vectors remain much less effective for therapeutic gene delivery than viral vectors.
Gene delivery technologies have markedly been improved in the past decades and this progress has been accompanied by a surge of gene therapy clinical trials. However, despite this significant progress, clinical experience with gene transfer technologies remains limited and clinical, manufacturing, regulatory, and commercialization hurdles are holding back progress in this field. Therefore, the field should continue to progress as it has over the past decade, cautiously and diligently. Many unanswered questions warrant further study. This Special Issue will cover recent advances in gene transfer technologies and in in vivo therapeutic applications of these technologies.
Prof. Dr. Bart De Geest
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 papers will be 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.
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- Gene therapy
- Gene transfer
- Hepatocyte-directed gene therapy
- In vivo gene therapy
- Ex vivo gene therapy
- Animal model