Welcome to the Konermann Lab!

The ability to perturb desired RNA molecules in a cell with high efficiency and specificity is a key technology across biomedical science. Despite rapid progress in effective technologies for genome engineering, analogous systems for transcriptome engineering lag behind their DNA counterparts. We recently identified the RNA-targeting type VI-D CRISPR-Cas family (Cas13d), in particular the engineered Cas13d from R. flavefaciens strain XPD3002 (CasRx) (Konermann et al., 2018), which are the smallest and most efficient Cas13 RNA targeting effectors in mammalian and plant cells reported to date. We are working to further characterize and optimize the Cas13d enzymes as RNA-targeting tools (Wei et al., 2022) to advance the investigation of RNA biology and application in RNA therapeutics.

High-throughput CRISPR perturbation screening has emerged as a powerful approach to interrogate gene expression networks (Konermann et al., 2015; Joung et al., 2017; Biering et al., 2022). We are developing and applying methods for CRISPR screening in stem cell-derived models of the human brain to investigate the cellular and molecular pathways driving genetic risk in neurodegenerative disease. Concurrently, we are developing computational methods for analyzing large-scale perturbation datasets towards the goal of elucidating the genetic architecture of complex disease risk.