Multiplex 'Conditional' Mice for Rapid and Affordable Pre-clinical Testing

 描述(由申请人提供)：小鼠癌症模型已被证明是测试新癌症疗法安全性和有效性的高效临床前模型。然而，产生最适用的癌症小鼠模型是昂贵、耗时的，因此没有得到广泛应用。利用最近开发的尖端技术生产新的癌症小鼠模型将以更负担得起和更快的方式生产出更好的模型，从而使新的治疗方法能够及时进入临床。为此，我们将优化和实施一项名为CRISPR系统的新技术。这一系统允许研究人员在特定细胞中一次特定地删除或修改小鼠基因组的多个部分，以诱导肿瘤发展。这种癌症发展模式作为临床前测试平台，比目前的模式更快，更具成本效益，更易于使用。这将允许许多实验室在准确的小鼠癌症模型上进行临床前测试，但由于前述缺点，这些实验室没有资源来利用当前的小鼠模型。我们建议使用CRISPR系统来生成快速发病的骨肉瘤和胰腺癌的新模型，这两种疾病分别影响儿童和成人患者。基于CRISPR的模型相对于传统模型的所有优势肯定会吸引许多研究实验室利用癌症研究和其他疾病领域的技术潜力。

 DESCRIPTION (provided by applicant): Mouse models of cancer have proven to be highly effective pre-clinical models for testing new cancer therapies for safety and efficacy. However, the generation of the most applicable mouse models of cancer is costly, time-consuming, and therefore not widely implemented. Generating new mouse models of cancer utilizing recently developed cutting-edge technologies will produce superior models in a more affordable and rapid fashion allowing new therapies to reach the clinic in a timely manner. To this end, we will optimize and implement a newly developed technology termed the CRISPR system. This system allows researchers to specifically delete or modify multiple portions of the mouse genome at one time in specific cells to induce tumor development. This model of cancer development as a pre- clinical testing platform is superior to current models as it is more rapid, cost effective, and easy to use. This will allow pre-clinical testing in accurate mouse models of cancer to be performed by many labs that do not have the resources to utilize the current mouse models, due to the aforementioned drawbacks. We propose to use the CRISPR system to generate new models of rapid onset osteosarcoma and pancreatic cancer, devastating diseases that affect pediatric and adult patients, respectively. All of the advantages of the CRISPR based models over traditional models will certainly attract many research labs to capitalize on the technologies potential in the field of cancer research and other diseases.