个人简历

学习经历 2010年12⽉-2014年05⽉05⽇ 丹麦奥胡斯⼤学 博⼠学位 导师：Jacob G. Mikkelsen 2009年08⽉-2010年07⽉ 瑞典卡洛琳斯卡医学院 博士生 导师：Thomas Perlmann (现诺奖委员会秘书长) 2006年09⽉-2009年07⽉ 中国科学院研究⽣院 硕⼠学位 导师：张红 2002年09⽉-2006年07⽉ ⼭东⼤学 本科学位 工作经历 2017.11-至今, 上海交通大学, 系统生物医学研究院, 研究员 2016.3-2017.11, 丹麦奥胡斯大学, 博士后, 合作导师: Soren Paludan 2014.1-2016.2, 丹麦奥胡斯大学, 博士后, 合作导师: Jacob G. Mikkelsen

研究领域

（1）利用实验室开发的新型CRISPR基因编辑递送技术，开展湿性老年黄斑变性、病毒性角膜炎等疾病基因治疗的临床前研究。（2）利用实验室开发的下一代的慢病毒载体技术，开展地中海贫血、镰刀型贫血等重大遗传疾病基因治疗的临床前研究。（3）开发新型溶瘤病毒技术，激活人体免疫系统，用于治疗实体瘤。

近期论文

1. Immunostimulatory guide RNAs mediate potent antiviral response; BioRxiv; Yujia Cai*, Alice Knudsen, Samuel Joseph Windross, Martin Kristian Thomsen, Søren Riis Paludan*. 2018. 2. Attenuation of cGAS-STING signaling is mediated by a p62/SQSTM1-dependent autophagy pathway activated by TBK1; EMBO J; 37(8); Prabakaran, T., C. Bodda., C. Krapp., B.C. Zhang., M.H. Christensen., C. Sun., L. Reinert., Y. Cai., S.B. Jensen., M.K. Skouboe., J.R. Nyengaard., C.B. Thompson., R.J. Lebbink., G.C. Sen., G. van Loo., R. Nielsen., M. Komatsu., L.N. Nejsum., M.R. Jakobsen., M. Gyrd-Hansen and S.R. Paludan*. 2018 3. In Vivo Knockout of the Vegfa Gene by Lentiviral Delivery of CRISPR/Cas9 in Mouse Retinal Pigment Epithelium Cells; Molecular therapy - Nucleic acids; 9: p. 89-99; Holmgaard, A., A.L. Askou., J.N.E. Benckendorff., E.A. Thomsen., Y. Cai., T. Bek., J.G. Mikkelsen and T.J. Corydon*. 2017 4. Inborn errors in RNA polymerase III underlie severe varicella zoster virus infections; J Clin Invest; 127(9): p. 3543-3556; Ogunjimi, B., S.Y. Zhang., K.B. Sorensen., K.A. Skipper., M. Carter-Timofte., G. Kerner., S. Luecke., T. Prabakaran., Y. Cai., J. Meester., E. Bartholomeus., N.A. Bolar., G. Vandeweyer., C. Claes., Y. Sillis., L. Lorenzo., R.A. Fiorenza., S. Boucherit., C. Dielman., S. Heynderickx., G. Elias., A. Kurotova., A.V. Auwera., L. Verstraete., L. Lagae., H. Verhelst., A. Jansen., J. Ramet., A. Suls., E. Smits., B. Ceulemans., L. Van Laer., G. Plat Wilson., J. Kreth., C. Picard., H. Von Bernuth., J. Fluss., S. Chabrier., L. Abel., G. Mortier., S. Fribourg., J.G. Mikkelsen., J.L. Casanova., S.R. Paludan* and T.H. Mogensen*. 2017 5. Targeted, homology-driven insertion in stem cells by ZFN-loaded 'all-in-one' lentiviral vectors; eLIFE; 5; Cai, Y., Laustsen, A., Zhou, Y., Sun, C., Anderson, M.V., Li, S., Uldbjerg, N., Luo, Y., Jakobsen, M.R. and Mikkelsen, J.G.* 2016 6. Lentiviral delivery of proteins for genome engineering; Current Gene Therapy; 16, E-pub ahead of print; Cai, Y. and Mikkelsen, J.G.* 2016 7. Lentivirus pre-packed with Cas9 protein for safer gene editing; Gene Therapy; 23(7), 627-633; Choi, J.G., Dang, Y., Abraham, S., Ma, H., Zhang, J., Guo, H., Cai, Y., Mikkelsen, J.G., Wu, H., Shankar, P., Manjunath N.* 2016 8. Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases; eLIFE; 3, e01911; Cai, Y., Bak, R.O. and Mikkelsen, J.G.* 2014 9. Driving DNA transposition by lentiviral protein transduction; Mobile Genetic Elements; 4, e29591; Cai, Y., Mikkelsen, J.G.* 2014 10. DNA transposition by protein transduction of the piggyBac transposase from lentiviral Gag precursors; Nucleic Acids Research; 10.1093/nar/gkt1163; Cai, Y., Bak, R.O., Krogh, L.B., Staunstrup N.H., Moldt B., Corydon T.J., Schr_der L.D., Mikkelsen J.G.* 2014 11. Efficient sleeping beauty DNA transposition from DNA minicircles; Molecular therapy - Nucleic acids; 2, e74; Sharma, N., Cai, Y., Bak, R.O., Jakobsen M.R., Schr_der L.D., Mikkelsen J.G.* 2013 12. A lentiviral vector-based genetic sensor system for comparative analysis of permeability and activity of vitamin D3 analogues in xenotransplanted human skin; Experimental Dermatology; 22, 178-183; Staunstrup, N.H., Bak, R.O., Cai, Y., Svensson, L., Petersen, T.K., Rosada, C., Stenderup, K., Bolund, L. and Mikkelsen, J.G.* 2013