Paula M. Cannon

This study engineers B cells to express fully customizable antibodies with enhanced Fc functions through genome editing of the immunoglobulin Heavy chain locus.

This study shows that knocking down astrocytic MCT4 in the motor cortex impairs motor learning and reduces dendritic spines and neuronal activity.

This study optimizes AAV6 transduction for ex vivo genome editing of human lymphocytes, achieving higher editing rates with reduced vector usage and toxicity.

The paper evaluates clade F AAVs for homologous recombination-based genome editing without DSBs, finding no detectable editing without ZFNs.

This study shows that inhibiting PIKFYVE kinase rescues both gain- and loss-of-function C9ORF72 disease mechanisms in mouse models of ALS/FTD.