Hildegard Büning, PhD

At-Large Director (2021-2024)

I am fascinated by the concept of gene and cell therapy. I am convinced that gene and cell therapy will permanently change the face of medicine. With my own research as well as with my engagement in the various gene therapy societies and beyond, it is my desire to help bring gene and cell therapy to patients in need. Safe and efficient gene delivery tools are key requirements for the success of these treatment strategies. I have dedicated my scientific career to work on adeno-associated viruses (AAV) and the vectors derived from them. AAV vectors are widely used and even first generation AAV vectors were shown to deliver impressive clinical benefit as in vivo gene therapies for various diseases. However, in order to unleash the full potential of AAV vectors, some challenges remain to be overcome, such as recognition by the host immune system, the broad tropism of natural AAV serotypes, and the need for high vector doses. My research group seeks to address these key limitations by two interconnected approaches: 1) in-depth analysis of AAV infection biology and cell responses elicited upon cell infection or transduction, and 2) the development of engineering strategies that address the identified pre- and post-entry barriers. We use these strategies to develop specifically tailored vectors that will serve as optimized gene transfer tools for the next generation of in vivo gene and cell therapies.