Results for Gene Therapy

Dr. George's basic and clinical research interests are in the development of novel therapeutics for hemophilia. Her basic science laboratory studies the molecular basis of coagulation, and she is the principal investigator of ongoing hemophilia A and B gene therapy trials.

Dr. Margaritis uses biochemical, molecular, and complex in vivo methodology within the field of coagulation to advance the understanding of molecular mechanisms involved in pro- and anti-coagulant reactions, and translate research for the treatment of coagulation defects.

The research in the Sabatino Laboratory is focused on hemophilia, an inherited bleeding disorder. The interests of the laboratory include the study of variants of coagulation factor VIII to understand the biochemical properties of these proteins and to identify novel variants with enhanced function, and the development of gene-based therapeutic approaches for treating hemophilia.

Dr. Weitzman's research program aims to understand host responses to virus infection, and the cellular environment encountered and manipulated by viruses. He studies multiple viruses in an integrated experimental approach that combines biochemistry, molecular biology, genetics, and cell biology.

Dr. Davidson works to understand the molecular basis of childhood onset neurodegenerative diseases and the development of gene and small molecule therapies for treatment. She also focuses on how noncoding RNAs participate in neural development and neurodegenerative disease processes, and how they can be harnessed for therapies.

Dr. Flake is a general pediatric surgeon with a clinical and research focus on prenatal treatment ranging from the fetal surgical repair of anatomic anomalies to prenatal stem cell and gene therapy. He has extensive experience in developing rodent, canine, and sheep models for in utero transplantation and for investigating fetal surgery for anatomic malformations.

Dr. Chorny's research program focuses on nanomedicines as well as drug, cell, and gene delivery systems for treating proliferative disease. His current research specifically focuses on the use of nanoparticle-mediated delivery to solid tumors and site-specific delivery for cardiovascular disease.

As a physician-scientist, Dr. Bernt's goal is to further the understanding of the role of transcriptional regulation in pediatric hematopoietic stem cell biology and leukemia, and translate findings into novel therapies.

Dr. van der Loo is an Adjunct Associate Professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania and Director of the Clinical Vector Core at CHOP, focusing on manufacturing of GMP and GMP process-comparable AAV and Lentiviral vectors for preclinical and gene therapy early phase clinical studies.

Dr. Bhoj's genetics research aims to discover new human disease genes, their mechanisms, and potential targeted therapies. In addition to ongoing gene discovery efforts, Dr. Bhoj focuses on three novel genes that lead to pediatric neurologic dysfunction: TBC1 domain-containing kinase, Histone 3.3 (H3F3A and H3F3B), and MAP4K4.