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Advancing CRISPR with deep learning: A comprehensive review of models and databases
Roghayyeh Alipanahi1, Leila Safari1, Alireza Khanteymoori2
1Department of Computer Engineering, University of Zanjan, Zanjan, Iran.
Molecular Therapy. Nucleic Acids
|September 29, 2025
Summary
Predicting CRISPR gene editing efficiency is crucial for therapy development. Machine learning and deep learning tools, alongside comprehensive CRISPR databases, are key to improving prediction accuracy and minimizing off-target effects.
Area of Science:
- Genetics and Genomics
- Bioinformatics
- Biotechnology
Background:
- CRISPR technology offers powerful targeted genome editing capabilities.
- Off-target effects are a major hurdle limiting the clinical application of CRISPR gene editing therapies.
- Accurate prediction of CRISPR editing efficiency is vital for advancing gene-editing therapies.
Purpose of the Study:
- To review available CRISPR databases for enhancing the prediction of CRISPR on-target and off-target activity.
- To highlight the role of machine learning (ML) and deep learning (DL) in improving CRISPR efficiency predictions.
- To address the need for large, searchable databases to support ML/DL approaches in CRISPR research.
Main Methods:
- Review of existing literature on CRISPR databases.
- Analysis of machine learning and deep learning methodologies for predicting CRISPR activity.
- Discussion of sequence features and their incorporation into prediction models.
Main Results:
- Current prediction accuracy for CRISPR on-target and off-target activity is limited by training data availability.
- Machine learning and deep learning are emerging as leading methods for CRISPR efficiency prediction.
- The development of comprehensive and searchable CRISPR databases is essential for advancing ML/DL prediction tools.
Conclusions:
- Improved prediction of CRISPR efficiency is critical for the safe and effective clinical application of gene-editing therapies.
- Machine learning and deep learning approaches, supported by extensive databases, show significant promise in overcoming CRISPR's off-target challenges.
- Further development and integration of sequence features into DL models are expected to enhance prediction accuracy and clinical utility.
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