教育经历

2004-2009，中国科学院北京基因组研究所，生物信息学，博士

1999-2003，苏州大学，生物技术，学士

工作经历

2026-至今，昌平实验室，领衔科学家

2018-2026，西湖大学生命科学学院，特聘研究员

2014-2017，芝加哥大学人类遗传学系，Staff Scientist

2010-2014，芝加哥大学基因组与系统生物学研究所，博士后

2009-2009，中国科学院遗传与发育生物学研究所，访问学者

研究简介

围绕基因与细胞治疗新药研发中递送效率、表达可控性及体内细胞重编程等关键科学与技术挑战，系统开展新型药物递送体系、可调控基因表达与基因编辑策略以及体内免疫细胞编程等研究。项目组旨在推动功能基因组学研究向可转化、可落地的创新平台延伸，在真实疾病与治疗场景中探索和实践 AI4Science，不仅形成具有自主知识产权的技术体系，也为基因与细胞治疗的临床转化和产业化提供持续的科学支撑与创新动力。

代表性成果

1）Z. Lu*, K. Ni*, W. Liu*, Q. Song, R. Zheng, M. Wei, Y. Zhang, J. Wang, L. Wei, C. Wu, Q. Zhang, J. Wu, R. Zheng, S. Ding, C. Cheng, Y. Cong, Y. Xu, B. Kong, S. Wu, G. Wang, X. Wang, Y. Wang, X. Qian, R. Deng, H. Chen, Y. Li# & L. Ma#. AAV variant enables human T cell engineering in vivo. Vita, 2026

2）R. Zheng*, Z. Lu*, R. Wei*, Y. Shin*, J. Du*, Q. Zhang, J. Li, X. Wang, Y. Wei, B. Liu, Y. Chen, L. Ding, H. Zhang, H. Chen, J. Huang# & L. Ma#. Spacer Extension Restores Cleavage Activity by Enhancing PAM-Distal Interactions in High-Fidelity Cas9. Nature Structural & Molecular Biology, 2026.

3）Q. Song*, J. Wu*, Q. Zhang, S. Wu, X. Luo, M. Xu, Y. Xu, G. Wang, H. Yu, H. Chen, Z. Lu# & L. Ma#. Directed evolution of novel AAV variants using the MCMS library for enhanced CNS tropism and reduced liver targeting in mice. Molecular Therapy Methods & Clinical Development, 2025.

4）R. Wei*, Z. Yu*, L. Ding*, Z. Lu, K. Yao, H. Zhang, B. Huang, M. He# & L. Ma#. Improved split prime editors enable efficient in vivo genome editing. Cell Reports, 2024.

5）Y. Liu*, Z. Lu*, P. Wu*, Z. Liang, Z. Yu, K. Ni & L. Ma. The Transpeptidase Sortase A Binds Nucleic Acids and Mediates Mammalian Cell Labeling. Advanced Science, 2024.

6）H. Zhang*, J. Yan*, Z. Lu*, Y. Zhou, Q. Zhang, T. Cui, Y. Li, H. Chen & L. Ma. Deep sampling of gRNA in the human genome and deep-learning-informed prediction of gRNA activities. Cell Discovery, 2023.

7）Z. Yu*, Z. Lu*, J. Li*, Y. Wang*, P. Wu, Y. Li, Y. Zhou, B. Li, H. Zhang, Y. Liu & L. Ma. PEAC-seq adopts Prime Editor to detect CRISPR off-target and DNA translocation. Nature Communications, 2022.

8）Z. Lu*, K. Ni*, Y. Wang, Y. Zhou, Y. Li, J. Yan, Q. Song, M. Liu, Y. Xu, Z. Yu, T. Guo & L. Ma. An in-library ligation strategy and its application in CRISPR/Cas9 screening of high-order gRNA combinations. Nucleic Acids Research,  2022.

9）Q. Song*, K. Ni*, M. Liu*, Y. Li, L. Wang, Y. Wang, Y. Liu, Z. Yu, Y. Qi, Z. Lu & L. Ma. Direct-seq: programmed gRNA scaffold for streamlined scRNA-seq in CRISPR screen. Genome Biology, 2020.