Gene Therapy for Childhood Diseases
A special issue of Genes (ISSN 2073-4425). This special issue belongs to the section "Molecular Genetics and Genomics".
Deadline for manuscript submissions: 20 July 2024 | Viewed by 85
Special Issue Editor
Interests: gene therapy; AAV; pulmonology; animal models; large animal models; translational gene therapy
Special Issue Information
Dear Colleagues,
With new approaches to gene therapy for childhood diseases expanding at an astounding rate and making their way to clinics, including relatively new therapeutic modalities, such as gene editing, making there way to the clinics we want to highlight some of the original research going into translational programs for a variety of diseases that affect children worldwide.
We realize that obtaining a gene therapy product approved for clinical use requires many steps from the bench to the bedside stage. We invite manuscripts that explore the transition of animal models through to clinical trials.
Dr. Alisha M. Gruntman
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. 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
- rare diseases
- gene therapy
- gene editing
- animal models
- government approval
- neonatal testing
- neonatal delivery
- patient screening
- patient recruitment
