Asst Professor In Residence
M_Surgery
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.
Examples include:
-Engineering red blood cells to deliver novel protein payloads