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Providing a foundation for basic research necessary to assure the success of gene therapy.

Given all the developments in molecular genetics, the isolation and cloning of genes is now a relatively common procedure. Research now centers on somatic gene therapy, referring to the techniques used to insert a functioning gene into the somatic (non-reproductive) cells of a patient to correct an inborn genetic error or to provide a new function to the cell. Having individual genes available opens the way for gene therapy to take place. And yet, after an initial period of about six years of preclinical work and another thirteen years involving clinical trials, effective gene delivery still remains one of the central challenges in the field.

The Gene Therapy Program of the University of Pennsylvania comprises basic scientific research and core lab research services. Our focus is on developing effective gene vectors derived from recombinant viruses. Much of our current effort is in the development of new adeno-associated virus (AAV) vectors, although some of our research involves both adenoviruses and lentiviruses. Several basic science core laboratories work together to support the development of new vectors.

Contact: Gene Therapy Program Suite 2000, Translational Research Laboratories (TRL) 125 S. 31st Street Philadelphia, PA 19104-3403 Phone: 215-898-0226 Fax: 215-494-5444 GTP@mail.med.upenn.edu

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