Advances in Gene Therapy: Novel Vectors and Strategies
A special issue of Genes (ISSN 2073-4425). This special issue belongs to the section "Molecular Genetics and Genomics".
Deadline for manuscript submissions: closed (25 February 2024) | Viewed by 2123
Special Issue Editors
Interests: adeno-associated virus; gene therapy; gene editing; lysosomal storage diseases; CRISPR
Interests: enzyme replacement therapy; small molecule chaperones; gene therapy; gene editing
Special Issue Information
Dear Colleagues,
Background and history of this topic: Gene therapy has the great potential to treat numerous diseases and has thus attracted attention from academia and industry in the past decade, evidenced by the performance of hundreds of on-going clinical trials and multiple high-profile regulatory approvals.
Aim and scope of this Special Issue: In order to further enhance the safety and efficacy of gene therapies, extensive efforts have been made to formulate novel vectors and treatment strategies, ranging from viral capsid evolution, RNA editing, to on-target gene-editing strategies.
Cutting-edge research: Cutting-edge research in this field includes the following: novel engineered AAV capsids with enhanced tropism, gene-editing tools (base editing, prime editing, RNA editing, etc.), non-viral delivery methods (LNP, liposomes, exosomes, etc.), novel treatment strategies (tRNA, small activating RNA, split AAV systems, novel transgene expression cassette design, etc), and innovations in the clinical manufacturing of gene therapies.
The kind of papers we are soliciting: This Special Issue welcomes the submission of review articles, short communications, or full-length research articles discussing novel methodologies, vector (non-viral and viral) development, and therapeutic approaches that have broad impacts in the field of gene therapy.
Dr. Li Ou
Dr. Michael Przybilla
Dr. Xin Chen
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. Genes 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 2600 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
- AAV
- viral vectors
- non-viral vectors
- gene therapy
- gene editing
- LNP
- tRNA
