Biomolecular Systems Engineering - NCCR MSE

Biomolecular Systems Engineering

Our work is grounded on using in vivo genome editing, based on the CRISPR-Cas9 technology, to rapidly model disease-associated genetic mutations and causally link them to complex genetic disorders, such as cancer and autism spectrum disorder.

In this project, we are taking a multifaceted approach, combining genome editing, synthetic biology, and functional genomics, to test the causality of and phenotypically characterize disease-associated genetic mutations on a massive scale in vivo. Once we identify causal genetic variants and establish disease models we will develop novel therapies using genomic and cell-based methodologies.


F. Schmidt, M. Y. Cherepkova, R. Platt “Transcriptional recording by CRISPR spacer acquisition from RNA“ Nature 2018. [DOI]
G. Wang, R. D. Chow, L. Ye, C. D. Guzman, X. Dai, M. B. Dong, F. Zhang, P. A. Sharp, R. Platt, S. Chen “Mapping a functional cancer genome atlas of tumor suppressors in mouse liver using AAV-CRISPR–mediated direct in vivo screening“ Sci. Adv. 2018. [DOI]
R. D. Chow, C. D. Guzman, G. Wang, F. Schmidt, M. W. Youngblood, L. Ye, Y. Errami, M. B. Dong, M. A. Martinez, F. Zhang, P. Renauer, K. Bilguvar, M. Gunel, P. A. Sharp, R. Platt, S. Chen “AAV-mediated direct in vivo CRISPR screen identifies functional suppressors in glioblastoma“ Nat. Neurosci. 2017. [DOI]
F. Schmidt, R. Platt “Applications of CRISPR-Cas for synthetic biology and genetic recording“ Curr. Opin. Syst. Biol. 2017, 5:9-15. [DOI]
R. Platt, Y. Zhou, I. M. Slaymaker, A. S. Shetty, N. R. Weisbach, J. Kim, J. Sharma, M. Desai, S. Sood, H. R. Kempton, G. R. Crabtree, G. Feng, F. Zhang “Chd8 Mutation Leads to Autistic-like Behaviors and Impaired Striatal Circuits“ Cell Rep. 2017, 19:335-50. [DOI]