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TopicGene editing: delivery, innate immune sensing and genome screening

LecturerYujia Cai, Ph.D. Department of Biomedicine, Aarhus University, Denmark  

HostPro. Zhang Zhuohua

TimeAug 26, 2016 Fri 15:00

Add: F5 Auditorium National Key Laboratory of Medical Genetics, Xiangya Old Campus

Synopsis:

The gene editing tools such as ZFN, TALEN and CRISPR are rapidly re-shaping the translational medicine and basic biology. Oneof the remaining of obstacles to apply gene editors in gene therapy is delivery. We have engineered lentiviral vector to be a protein carrier – not a gene carrier as its traditional role – to ferry gene-modifying enzymes including ZFN, TALEN and Cas9. To retarget lentiviral vector for safe gene therapy, we further package a whole gene repair kit – ZFNs and donor sequence – into the same lentiviral particle and demonstrate targeted gene integration into specific loci in CD34+cells and iPSCs with no detectable off-targeting events. To apply CRISPR in gene therapy, it is crucial to understand how our immune system looks at the bacteria-derived nucleic acids. I will report sensing of gRNA by host RNA sensors. Lastly, I will briefly show our ongoing work to adopt CRISPR library to study the interactions between herpes simplex virus and neurons.

Biography:

Yujia Cai obtained BS at Shandong University and MS at Institute of modern physics, Chinese Academy of Sciences. Then he furthered his research at Karolinska Institute, Sweden and pursued his PhD at Aarhus University. His research areas: 1) Development of gene therapy include virus vector and gene editing tools; 2) Solve immunological problem by using gene editing tools. He has published 2 papers on eLIFE, and other related works on Nucleic Acids Research, Current Gene Therapy, Gene Therapy etc.

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