肿瘤细胞治疗研究
英文原题:Rising Star Engineering the Genome for Curative Futures.
Rising Star Engineering the Genome for Curative Futures.
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作为华东师范大学生命科学学院生物医学教授,我致力于开发先进基因组编辑技术,用于疾病建模和精准基因治疗。在湖南师范大学和德州农工大学接受的基础训练,使我对利用工程化细胞和动物模型理解人类疾病产生浓厚兴趣。自2013年以来,我在华东师范大学的实验室率先利用TALEN和CRISPR/Cas9快速构建用于疾病研究的敲除小鼠和大鼠模型。进入基因组编辑领域后,我转而专注于推进编辑工具,并为遗传病和癌症开发基因治疗策略。团队已开发出一系列高性能碱基编辑器,可作用于核DNA、线粒体DNA和RNA,在拓展编辑能力的同时提高精准性和安全性。利用这些技术,我们设计了多种治疗策略,并在遗传病细胞及动物模型中验证其疗效。通过合作,我们成功推动基因组编辑进入临床应用,为地中海贫血患者治疗作出贡献。
此外,我们还开发了用于淋巴瘤治疗的非病毒定点整合CAR-T 平台。展望未来,我希望开发新一代长片段定点整合技术并加速临床转化,让更多患者获得变革性治愈。
As a professor of biomedicine in the School of Life Sciences at East China Normal University (ECNU), I am dedicated to developing advanced genome editing technologies for disease modeling and precise gene therapy. My foundational training at Hunan Normal University and Texas A&M University cultivated a deep interest in using engineered cellular and animal models to understand human diseases. Since 2013, my laboratory at ECNU has pioneered the use of TALEN and CRISPR/Cas9 for the rapid generation of knockout mouse and rat models for disease studies.
Once stepped in genome editing field, I shifted my focus to advancing editing tools and developing gene therapy strategies for genetic disorders and cancer. My team has developed a suite of high-performance base editors for nuclear DNA, mitochondrial DNA, and RNA, broadening editing capabilities while enhancing precision and safety.
Leveraging these technologies, we have designed several therapeutic strategies that have shown efficacy in cellular and animal models of genetic diseases. Through collaborative efforts, we have successfully translated genome editing into clinical applications, contributing to the treatment of patients with -thalassemia.
Additionally, we have developed a non-viral, site-specific CAR-T platform for lymphoma therapy. Looking forward, I aim to develop the next generation of long-fragment, site-specific integration technologies and accelerate clinical translation to bring transformative cures to more patients.
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