Advances, Pitfalls and Future Perspectives for CRISPR/Cas9 Mediated Genome Editing 4.0

Dear Colleagues,

The 2020 Nobel Prize in Chemistry was awarded to Emmanuelle Charpentier and Jennifer A. Doudna “for the development of a method for genome editing”. Since the initial publication on the mechanism of CRISPR/Cas9 nuclease cleavage activity and its application for the direct targeting of genomic sequences “in vitro” in 2012, multiple parallel studies in different organisms (from bacteria to human cells) have proven its unparalleled efficiency as genetic “molecular scissors” for “in vivo” manipulations. Within a short period of time, CRISPR/Cas9-mediated genome editing has emerged as a state-of-the-art approach for precise gene modification in all model organisms. It has become a powerful technology for the generation of custom-designed gene variants and gaining molecular insights into different biochemical pathways or human diseases at the cellular or organism level. Moreover, CRISPR/Cas9-mediated genome editing is currently under intensive development for application in the field of human gene therapy and has recently been approved for its first clinical trials. However, despite the many advantages and great potential for future research and clinical applications, existing problems (off-target activity, the induction of genome rearrangements, cellular mosaicism in transgenes, etc.) still impede the full-scale use of the technology in biomedical research. Further efforts are needed to overcome these hurdles. This Special Issue aims to cover all areas of molecular-based research that use CRISPR/Cas9 and alternative CRISPR systems for genome editing and other applications in cellular or animal models. It welcomes original research, reviews and short communication articles of which CRISPR technology is the main topic or the tool for answering mechanistic molecular questions. This includes, but is not limited to, methodological advances and identified pitfalls in CRISPR-based technology, and its application to functional genomics for disease and human gene therapy, epigenomics, proteomics, RNA biology, systems biology, etc.

• Molecular insights into different approaches and methods for CRISPR/Cas9 genome editing;
• The generation of disease models;
• The CRISPR/Cas9-mediated genome editing of ES cells;
• The CRISPR/Cas9 system in cancer biology;
• CRISPR/Cas9-mediated genome editing in model organisms (plants, fungi and animals);
• The CRISPR/Cas9 system in epigenetic research;
• The CRISPR/Cas9 system in gene therapy—applications and the challenges in the implementation of this technology;
• The CRISPR/Cas9 system beyond genome editing (molecular markers, biosensors, etc.).

Dr. Alexander Kondrashov
Dr. Timofey S. Rozhdestvensky
Guest Editors

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• molecular insights into different approaches and methods for CRISPR/Cas9 genome editing
• the generation of disease models
• the CRISPR/Cas9-mediated genome editing of ES cells
• the CRISPR/Cas9 system in cancer biology
• CRISPR/Cas9-mediated genome editing in model organisms (plants, fungi and animals)
• the CRISPR/Cas9 system in epigenetic research
• the CRISPR/Cas9 system in gene therapy—applications and the challenges in the implementation of this technology
• the CRISPR/Cas9 system beyond genome editing (molecular markers, biosensors, etc.)