于晨课题组 
基因组工程 | 基因治疗

RESEARCH

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Our research group is interested in developing technologies for genome engineering and gene therapy. Using these tools we are eager to understand how genome functions during cancer development, and design new approaches to read, modulate, and engineer genome for disease treatment.

 

PRECISION GENOME ENGINEERING

Fundamentally, life is information transfer. Genome transfers the information it carries dynamically and at multiple scales simultaneously. We apply functional genomics approaches to interrogate the dynamic genome function in cell biology. For example, we developed an integrative screening approach based on CRISPR activation. Using this platform, we systematically interrogated the roles of transcription factor interactions on cell fate decision. By engineering genome function ranging from nucleic acid sequence to chromatin structure, we hope to unravel new information about cancer development and to offer more innovative tools for biology discovery.


NEXT-GENERATION GENE THERAPY

For gene therapy, we design tools for the treatment of genetic disease. We combine genome engineering with genetic circuits to develop such tools. For example, we developed efficient approaches to precisely edit the genome of human cells. We designed and engineered cells with advanced genome perturbation technologies to precisely control cell fate. Going forward, we will integrate synthetic control devices, genome perturbation circuits, and cell fate engineering into the next-generation gene therapies, and hope some of them will transform future medicine.


课题组致力于为基因组工程和基因治疗开发新型技术和工具。通过这些工具的应用,我们期望揭示基因组在疾病中行使其功能的机制;并通过对基因组功能的调控,设计开发新型的基因疗法。

1精准基因组工程。信息传递是一切生命现象的基础。基因组在多个尺度上动态传递本身所携带的信息,以决定细胞的性状和生命体的表型。我们采用功能基因组学方法研究基因组在决定细胞性状,特别是肿瘤中的功能。例如,我们开发了一个基于CRISPR activation的流程,系统研究了转录因子间相互作用对细胞命运的影响。通过多尺度上对基因组功能的调控,我们希望能够寻找癌症发生、发展与转移的新机制、新标志物和治疗靶点,并为生物学研究设计开发新的功能基因组学工具。

2新一代基因疗法。课题组还致力于为治疗遗传性疾病设计开发新型基因疗法。例如,我们开发了能够高效编辑人基因组的方法。我们利用CRISPR技术,设计了一个能够精准调控细胞命运的平台。我们将结合基因组工程方法与遗传回路原件,设计开发新型基因治疗工具。新工具将通过对基因组的调控,精准控制靶细胞命运,从而逆转细胞损伤、改善细胞功能,为遗传性疾病提供新的治疗方法。