Division of Translational Medicine and Human Genetics Research

David Fajgenbaum, MD, MBA, MSc, an assistant professor of Medicine at the Perelman School of Medicine at the University of Pennsylvania and associate director of patient impact in the Penn Orphan Disease Center, leads the Castleman Disease Research Program at Penn.

The Gene Therapy Program of the University of Pennsylvania focuses on developing effective gene vectors derived from recombinant viruses.

Genetic Counselors in the Division of Translational Medicine and Human Genetics participate in research in many areas.

The Genome Editing is focused on the functional study of cardiovascular and liver disease associated gene mutations, the development of regenerative medicine and gene therapy, and establishing a robust method for the genetic manipulation of adult ductal liver stem cells.

IRAD is the International Registry of Acute Aortic Dissection.

The Division of Translational Medicine and Human Genetics works closely with the Penn Medicine Orphan Disease Center.

Pharmacogenomics involves the study of how genetic variants influence response to medications. Many powerful drugs are available for the treatment of heart disease, cancer and other illnesses. While most medications work well for most people, some people fail to respond or suffer from serious sides effects. A significant portion of this variability can be explained by an individual’s genetic profile.

The Translational Genome Editing lab is focused on establishing robust CRISPR-mediated methods for the genetic manipulation of human-induced pluripotent stem cells (hiPSC) and the newly discovered human liver-derived ductal stem cells. We use human liver-derived ductal stem cells to study liver regenerative medicine and liver gene therapy, as well as cardiovascular and liver disease-associated gene mutations.