Liao Xichen, Liu Qi, Chuai Guohui
Department of Hematology, Tongji Hospital, Frontier Science Center for Stem Cell Research, Bioinformatics Department, School of Life Sciences and Technology, Tongji University, No. 1239 Siping Road, Yangpu District, Shanghai 200092, China.
Shanghai Key Laboratory of Anesthesiology and Brain Functional Modulation, Clinical Research Center for Anesthesiology and Perioperative Medicine, Translational Research Institute of Brain and Brain-Like Intelligence, Shanghai Fourth People's Hospital, Frontier Science Center for Stem Cell Research, Bioinformatics Department, School of Life Sciences and Technology, Tongji University, No. 1239 Siping Road, Yangpu District, Shanghai 200092, China.
Brief Bioinform. 2025 May 1;26(3). doi: 10.1093/bib/bbaf293.
The rapid development of gene editing technology has revolutionized life science research and biotechnology applications. Prime editing, a precise gene editing tool, has shown promise in various applications, including disease research and therapeutic interventions. However, its suboptimal editing efficiency for extensive fragments and lack of predictive models have hindered its widespread adoption. Existing models exhibit low prediction accuracy and limitations, such as neglecting epigenetic factors that impact gene editing effects. To address these challenges, we developed PrimeNet, a novel prediction model that integrates significant epigenetic factors, including chromatin accessibility and DNA methylation. By incorporating data from multiple cell lines and introducing multiscale convolution and attention mechanisms, PrimeNet enhances the accuracy of predictions and generalization performance. Our results show that PrimeNet achieves a Spearman correlation coefficient of 0.94 and 0.82 on two datasets originated from HEK293T and K562 cell lines, respectively, outperforming existing models. This novel model has the potential to guide experimental design, enhance the success rate of gene editing, and reduce unnecessary experimental costs, thereby advancing the application of gene editing technology in genetic disease treatment and related fields.
基因编辑技术的快速发展彻底改变了生命科学研究和生物技术应用。碱基编辑作为一种精确的基因编辑工具,在包括疾病研究和治疗干预在内的各种应用中显示出了潜力。然而,其对大片段的编辑效率欠佳以及缺乏预测模型阻碍了它的广泛应用。现有模型的预测准确性较低且存在局限性,比如忽略了影响基因编辑效果的表观遗传因素。为应对这些挑战,我们开发了PrimeNet,一种整合了包括染色质可及性和DNA甲基化等重要表观遗传因素的新型预测模型。通过整合来自多个细胞系的数据并引入多尺度卷积和注意力机制,PrimeNet提高了预测准确性和泛化性能。我们的结果表明,PrimeNet在分别源自HEK293T和K562细胞系的两个数据集上的斯皮尔曼相关系数分别达到0.94和0.82,优于现有模型。这种新型模型有潜力指导实验设计,提高基因编辑的成功率,并降低不必要的实验成本,从而推动基因编辑技术在遗传疾病治疗及相关领域的应用。