Ex Vivo Gene Therapy for Genetic Diseases

Ex vivo gene therapy is an attractive approach to treating numerous genetic conditions through isolating a population of cells from a patient, engineering altered gene expression in those cells, and delivering them back into the patient’s body. This offers an efficient and targeted therapy while minimizing effects on other tissues and complications from the immune system. This has been applied to numerous models of genetic diseases and shows great promise for new, more effective treatments.

Human Gene Therapy is organizing a special issue focused on ex vivo gene therapy to highlight the recent advancements, present new findings, and discuss the future goals and directions of the field. We aim to invite a discussion of diseases for which ex vivo therapy is being/could be applied, mechanisms of ex vivo therapies, challenges and innovations in ex vivo therapy, and methods to improve delivery and efficacy of ex vivo gene therapy.

For this issue, we welcome submissions of review articles, research manuscripts, methods papers, and commentaries about ex vivo gene therapy.

Areas of interest include, but are not limited to:

• How ex vivo gene therapy is being applied to a specific condition or type of disease
• Cells/tissues that can be targeted for ex vivo gene therapy
• Mechanisms of ex vivo engineering
• Mechanisms of engineered cells interacting with host cells
• Methods for improved ex vivo gene therapy efficacy

We also encourage submission of papers that have been published in conference proceedings, with the requirement that the authors have made significant extensions as compared to the already published version of the study.

Manuscript Submission Deadline: September 30, 2023

When submitting a paper, please upload it under the manuscript type / category labeled Special Issue Submission: Ex Vivo Gene Therapy for Genetic Diseases

Questions or pre-submission queries? Please contact Guest Editors Alessandra Biffi and Brian Bigger or Managing Editor Thomas Gallagher.