While we have long known the location of disease-causing mutations in the genome, the discovery of CRISPR finally gave us the ability to correct these typos back to what they should be in healthy patients. While this effort has yielded novel therapies in the clinic, in my own lab I want to look beyond simply correcting DNA typos and instead use genome editing to introduce novel functions into cells for therapeutic purposes.
-Engineering red blood cells to deliver novel protein payloads
-Creating genome editing strategies that bias stem cell differentiation to produce clinically relevant cell types
-Engineering kill switches to prevent differentiation into unwanted cell types
-Developing novel ways to regulate therapeutic protein stability and expression using small molecules
-Multiplexing editing in order to introduce multiple genome editing events simultaneously (such as correcting a disease-causing mutation and adding a kill switch that could be activated in the case of an adverse event)
With special focus on hematopoietic stem cells and red blood cells, my main goal is to close the gap between synthetic biologists and clinicians in order to address current bottlenecks in treating the hemoglobinopathies and other blood disorders. While this is my current focus, the tools I am developing are cell type- and disease-agnostic and I am always open to expanding these concepts into new areas.