Genome Editing
Cas9 nuclease and derived technologies like base editors and prime editors have great potential to modify cellular genomes and treat genetic disease. However, many challenges still exist – editors need to be optimized for each target and characterized to identify the efficiency that is necessary to treat disease. In many cases, emerging delivery technologies will be required to reach a suitable efficiency threshold. For each candidate method, the risks of immunogenicity and off-target editing must be defined and minimized. We seek to develop new genome editing therapeutics to expand the benefits of editing tools to the maximal number of patients.
Gene Regulation
Cis-regulatory elements in the genome use a complex code to direct healthy gene transcription and is impacted by distal elements and chromatin context. We seek to study and define the phenomena that govern the transcription of disease-associated genes and thus learn to decipher and correct dysfunctions.
