About this Research Topic
Gene editing has become a staple for genetic manipulation in the laboratory and is central to the study of genes and their functions. As these tools become more powerful, flexible, and easy to use, the question of whether these methods can be utilized to treat human diseases is frequently raised. Unfortunately, there are many barriers to translating gene editing approaches to the clinic. Issues regarding immunogenicity, genotoxicity, and off-target editing events are just some of the challenges.
For more than two decades, gene delivery vectors that are based on viruses have been proven to be powerful and safe modalities for gene transfer. Many gene therapy viral vectors have been used successfully in clinical settings, while some have gained regulatory approval for commercial use. Thus, the combined fields of genome editing and viral vectorology are perfect mates in the pursuit of treating genetic diseases.
This article collection aims to highlight current advances in gene editing and compatible viral vector delivery systems. It will showcase the forefront of research that encompasses the fields of genome editing and viral vectorology, working in concert to tackle genetic and acquired diseases. The collection will address the challenges of directing gene correction using viral vectors with in vitro, in vivo, and ex vivo models for clinical translation. It will also address issues regarding the safety of delivering vector and gene editing components to patients. Finally, readers will gain an appreciation for the rich diversity of approaches developed to treat human disease, and how far the field has advanced gene editing/therapy systems.
This Research Topic welcomes contributions such as:
• Articles covering basic research findings that may directly or indirectly define the barriers for gene editing potency and vector transduction (i.e., immunology, genotoxicity, pathogenicity).
• Articles reporting on key advances in engineering novel vectors or gene editing platforms.
• Articles reporting proof-of-concept studies in relevant preclinical models.
• Articles that highlight paradigm-shifting concepts or techniques for gene editing and viral vectors.
• Methods articles for improving diagnostics for manufacturing or analytical tools for gauging gene editing potencies.
• Articles pertaining to the use of gene editing vectors for generating animal models or novel cell engineering approaches.
• Original review articles on genome engineering and viral vector development.
Dr. Dirk Grimm is a co-founder, shareholder, managing director and chief scientific officer of AaviGen GmbH. Dr. Dirk Grimm holds various patents on AAV vector technology.
Dr. Jeffrey Chamberlain is a co-founder of Ballard Biologics and of KineaBio, and he holds shares in both companies. Kinea is considering licensing a pending patent covering muscle-specific gene editing. Dr. Jeffrey Chamberlain is also on the Scientific Advisory Board of Solid Biosciences, a publicly traded company (SLDB) and he has equity in Solid. However, those are not doing genome engineering.
Dr. Phillip Tai is a co-inventor on patents related to gene therapy. Dr. Phillip Tai also holds grants with BridgeBio and CANbridge Pharmaceuticals and holds shares with AAVAA.
Keywords: Viral vectors, Gene editing, CRISPR-Cas, Rare disease, Gene therapy
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