AAVHSC: A Novel Genetic Medicine Platform for Gene Therapy & Genome Editing
January 21, 2021, 4:00 pm
Location: Join Zoom Event
Speaker: Saswati Chatterjee, PhD, City of Hope
A novel adeno-associated virus family derived from healthy human hematopoietic stem cells, the AAVHSCs, show remarkable potential for gene therapy and genome editing. Their widespread tissue tropism including the liver, muscle, heart and the retina and their ability to cross the blood brain barrier and transduce cells of the central nervous system makes them well-suited for systemic treatment of inherited and acquired diseases. Notably, AAVHSC mediate efficient and seamless homologous recombination-based genome editing without the requirement for nuclease-mediated double stranded DNA breaks. The potential of AAVHSC for therapeutic gene therapy and genome editing will be discussed.
PhD, City of Hope
Saswati Chatterjee, PhD, Professor, Department of Surgery, City of Hope has focused upon developing genetic strategies for the treatment of human diseases. Following her PhD at McGill University, she pursued post-doctoral fellowships at NIH before moving to the City of Hope. Her research focuses upon the mechanistic and translational aspects of AAV vectors for genetic medicines. More recently this work led to the discovery of interesting and unexpected genetic potentials of the AAVHSC platform. She is the scientific cofounder of Homology Medicines Inc, Bedford, MA, which is further developing the AAVHSC platform for gene therapy and genome editing.