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

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A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Biology".

Deadline for manuscript submissions: 30 April 2026 | Viewed by 19

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Special Issue Editor

Dr. Timofey S. Rozhdestvensky

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Guest Editor

Medical Faculty, Core Facility Transgenic Animal and Genetic Engineering Models (TRAM), University of Muenster, Von-Esmarch-Str. 56, D-48149 Muenster, Germany
Interests: genome editing; CRISPR-Cas9 technology; programmable DNA endonucleases; nervous system diseases; RNA biology; disease-associated RNAs; non-protein coding RNAs
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Special Issue Information

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

Manuscript Submission Information

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Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.

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Keywords

genome editing
CRISPR/Cas
disease modeling
functional genomics
organoids

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