Cellular and Gene Editing Research

Cellular Operations

We develop novel genome editing techniques, cellular differentiation and cellular phenotyping systems, especially with respect to high-throughput investigation of gene and non-coding regulatory element function in Alzheimer's and Parkinson's diseases. 

• Developing induced pluripotent stem cell models of genetic disease
• Development of high throughput single cell and spatial genomics technologies
  
• Genetic screening techniques using pooled and arrayed CRISPR/Cas9
• Differentiation protocols of pluripotent stem cells to brain and blood-derived lineages
• Application of screening to complex phenotypic readouts such as single cell transcriptomics or spatial genomics

We develop novel cutting-edge CRISPR screening techniques such as saturation mutagenesis, base editing, dual guide libraries and CRISPRa/i and couple these to complex phenotypic readouts such as single cell transcriptomics, spatial genomics or phenotypic assays of cellular function. We also work with and develop human cellular systems and differentiation protocols of human induced pluripotent stem cells (hiPSCs) in both 2D and 3D to better model human disease states in vitro. The systems developed will be applied to probe the genetic basis of cellular function in health and disease and understand the underlying cell biology.

We have a particular interest in the genetic causes of neurodegeneration, especially Alzheimer’s and Parkinson’s diseases and use some of these screening methodologies to identify the genes involved and understand the consequences of genetic mutations as part of two OpenTargets projects, Neuroinflammation and NeuroID.