AAV Gene Therapy: A novel approach to AAV biopanning in human brain

Gene therapy holds potential for treating neurological diseases by delivering genetic information into specific cell types. However, selective and efficient targeting of cell types remains challenging. Research into engineered adeno-associated virus (AAV) capsids shows promise but is impeded by inefficient models lacking relevant human cell physiology. Our project uses resected human brain tissues from various neurosurgical conditions to screen novel libraries of AAV capsid variants. With a multi-omic sequencing approach and through automated tissue imaging, we identify and optimize capsid variants for selective transduction across numerous diseases. This capsid library could deliver genetic information precisely into defined diseased cell types, opening new avenues for treating severe neurological diseases. We aim to create libraries of cell-type-specific AAV capsids, using our human brain model, to facilitate genetic information delivery into any desired cell type.

 

Learning Objectives