Genome Editing in Disease Research: Applications in Animal Models and Organoids

Dear Colleagues,

Genome editing has become a powerful approach for investigating disease mechanisms and developing therapeutic strategies. Advances in CRISPR/Cas systems, along with base and prime editing technologies, have increased the ability to generate precise and reproducible disease models. Animal models continue to play a crucial role in understanding systemic disease processes and evaluating therapeutic responses, while recent advances in organoid research provide powerful platforms for recapitulating the patient-specific physiology in vitro. Beyond accelerating scientific discovery, the integration of genome editing with organoid models contributes to the 3Rs—Replacement, Reduction, and Refinement—by offering alternatives to certain animal experiments, reducing experimental numbers, and refining research protocols. Together, these complementary systems expand our understanding of human pathologies and facilitate translational applications.

This Special Issue aims to highlight cutting-edge studies that employ genome editing to advance disease modeling, functional genomics, and therapeutic development. In addition, contributions that address the pitfalls, limitations, and practical solutions to improve the robustness and reproducibility of these technologies are strongly encouraged.

Research Topics:

• Development of CRISPR-based animal disease models
• Genome editing in organoid systems for modeling physiological and cellular processes
• Base and prime editing applications in functional genomics and gene therapy research
• Epigenome editing to investigate gene regulation and underlying disease mechanisms
• Advanced delivery strategies for efficient and precise genome editing in complex models
• Comparative studies integrating animal models and organoid systems
• Strategies to implement genome editing advancing the 3Rs principles
• Approaches to improve reproducibility, efficiency, and safety of editing technologies

Dr. Timofey S. Rozhdestvensky
Guest Editor

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