Special Issue

Gene Therapy for Genetic and Acquired Diseases: Current Progress and Future Directions

Submission Deadline: March 31, 2027 (Open) Submit Now

Guest Editor

Rina Kansal, MD

1. Molecular Oncology and Genetics, Diagnostic Laboratories, Versiti Blood Center of Wisconsin, Milwaukee, WI, United States
2. Department of Pathology and Anatomical Sciences, University at Buffalo, Buffalo, NY, United States

Website1 | Website2 | E-Mail

Research Interests: Precision medicine; hematopathology; hematology; oncologic pathology; molecular genetic pathology; genomics; molecular diagnostic pathology; cytogenetics

About This Topic

Gene therapy is transforming the treatment of inherited and acquired disorders. This Special Issue aims to provide an overview of current progress and highlight advances in gene therapy, including CRISPR-based genome editing and cell-based therapies, with a focus on clinical and translational applications.

We welcome reviews on therapies targeting monogenic diseases, hematologic disorders, malignancies, and other acquired conditions, particularly addressing challenges such as effective delivery, in situ strategies, and clinical translation of gene therapy platforms. Additional topics include safety and efficacy, vector innovation, and studies exploring novel targets and personalized approaches.

Keywords

Gene Therapy; CRISPR-Cas Systems; Gene Delivery Systems; RNA; Precision Medicine; Translational Medical Research; Genome Editing; Nanotechnology

Manuscript Submission Information

Manuscripts should be submitted through the LIDSEN Submission System. Detailed information on manuscript preparation and submission is available in the Instructions for Authors. All submitted articles will be thoroughly refereed through a single-blind peer-review process and will be processed following the Editorial Process and Quality Control policy. Upon acceptance, the article will be immediately published in a regular issue of the journal and will be listed together on the special issue website, with a label that the article belongs to the Special Issue. LIDSEN distributes articles under the Creative Commons Attribution (CC BY 4.0) License in an open-access model. The authors own the copyright to the article, and the article can be free to access, distribute, and reuse provided that the original work is correctly cited.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). Research articles and review articles are highly invited. Authors are encouraged to send the tentative title and abstract of the planned paper to the Editorial Office (genetics@lidsen.com) for record. If you have any questions, please do not hesitate to contact the Editorial Office.

Welcome your submission!