科学研究
基因治疗课题组
  课题组长期从事基因治疗载体、基因编辑递送技术的研发以及基因治疗载体与机体免疫系统互作的研究,致力于新技术的临床转化。领导了国际首个在体的CRISPR抗病毒基因治疗临床研究。开发了慢病毒蛋白转导以及mRNA转导的PCT国际专利技术,并实现转化应用。近年来,论文以通讯或第一作者发表在Nature Biotechnology, Nature Biomedical Engineering, eLIFE, Nucleic Acids Research, Gene Therapy等期刊。担任Nature Biotechnology等期刊的审稿人。研究成果被《人民日报》、 《新华社》等权威媒体报道。联合创立本导基因,获数亿的资金支持。所培养的2位博⼠获得博⼠⽣国家奖学⾦和上海市优秀毕业生。其中,凌思凯博士获得2021年度上海交通大学优秀博士论文奖(生物学唯一入围者)。

课题组长:蔡宇伽

课题组成员

工作人员乐黄莺  张雪  段文博(博士后)

学生:博士:尹笛   戴瑶   钟亦晔  姜卓凡

          硕士:张淑慧  陆斯瑽  郭钰  夏子媛  张洛妮

 

主要研究方向

1.发现:利用CRISPR全基因组文库、单细胞测序等系统性工具发现药物新靶点和生物学新机制。

2.发明:发明新型基因编辑工具、新型递送载体、新型免疫治疗技术,实现遗传疾病、肿瘤、病毒感染等难治性重大疾病的治疗。

3.应用:将原创性的发现和发明用于临床治疗,开发全球意义的first in class药物。

 

代表性成果
1. Targeting herpes simplex virus with CRISPR-Cas9 cures herpetic stromal keratitis in mice; Nat Biotechnol;39(5):567-577; Yin,D ., Ling,S., Wang,D.,Dai,Y., Jiang,H., Zhou,X ., Paludan ,S.R. , Hong,J. ,Cai,Y.*2021

2. Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice; Nat Biomed Eng; 5(2):144-156; Ling,S., Yang,S., Hu,X., Yin,D.,Dai,Y., Qian,X., Wang,D., Pan,X., Hong,J., Sun,X., Yang,H., Paludan,S.R., Cai,Y.*2021

3. An overview of development in gene therapeutics in China; Gene Ther; 27(7-8):338-348; Wang,D.,Wang,K.,Cai,Y.*2020

4. 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

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. 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

7. Lentiviral delivery of proteins for genome engineering; Current Gene Therapy; 16, E-pub ahead of print; Cai, Y. and Mikkelsen, J.G.* 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. 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., Schrøder L.D., Mikkelsen J.G.* 2014
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