Assessment and modulation of immune response to gene editing reagents and delivery vehicles

Gene editing-based technologies hold tremendous promise for permanent correction of monogenic diseases by repairing disease causing mutations. However, immune response against delivery modalities poses a major challenge to efficient editing and persistence of edited alleles in the patient population. The precise nature of immune response to gene editors and delivery platforms remain poorly understood.

In our lab, we are exploring the immune response to Cas9 nucleases, delivered either with Adeno Associated Viruses (AAVs) or lipid nanoparticles (LNPs). We are also interested in exploring immune modulating strategies to overcome host immune responses against gene editing reagents delivered using viral and non-viral modalities.