Gene Therapy for Hemophilia

The Center for Cellular and Molecular Therapeutics facilitates rapid translation of pre-clinical discoveries into clinical application. One of few such programs based at a pediatric institution, the center collaborates with other major programs to pursue new therapies for inherited and acquired disorders. Katherine A. High, MD, is the director of the Center for Cellular and Molecular Therapeutics at CHOP. 

Current research includes:

Gene therapy for hemophilia

Dr. High has used adeno-associated virus (AAV) vectors in clinical trials of gene therapy for hemophilia B. Dr. High also published a study in Nature of genome editing technology in 2011 that was the first successful demonstration of genome editing to correct a genetic defect in vivo.


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