 |
 |
 |
 |
 |
 |
 |
Member Information
James M. Wilson, MD, PhD
John Herr Musser Professor and Chair
Department of Molecular and Cellular Engineering
Office Phone: 215-898-3000
Office Fax: 215-898-6588
Email: wilsonjm@mail.med.upenn.edu
Website(s):
Education:
Keywords: None available
Research and/or Clinical Interests:
None available.
Summary:
Dr. Wilson's research focuses on the development of somatic gene therapies in the treatment of a variety diseases, both inherited and acquired. Another important emphasis is in disease pathogenesis. A critically important model to this program has been gene transfer to the liver for treatment of dyslipidemias that lead to premature coronary artery disease. Dr. Wilson's laboratory was the first to perform ex vivo gene therapy for liver in the treatment of familial hypercholesterolemia and more recently, has developed in vivo approaches for transferring genes to liver the modulate lipoprotein metabolism.
Another important initiative in the lab is targeting genes to skeletal and cardiac muscle using vectors based on DNA viruses. It was discovered that adeno-associated virus is an effective vector for efficiently transferring genes to cardiac muscle. This is accomplished without eliciting humoral or cellular immune responses to the transgene product. Applications of this technology are being considered for the treatment of hypertrophic cardiomyopathy and revascularization in ischemic heart disease.
Additionally, Dr. Wilson's laboratory focuses on the use of somatic gene transfer for studying and potentially treating inheritive neuromuscular disorders. They have characterized the basic biology of DNA viral based vectors in the study of gene transfer to neuron and muscle fibers. Interesting results with respect to eliciting immune responses to transgene products have surfaced. These strategies are being tested in preclinical models for the treatment of a number of disorders including Limb Girdle and Duchennes muscular dystrophy.
Representative Publications:
Yang Y and Wilson JM. CD40 Ligand-Dependent T Cell Activation: Requirement of B7-CD28 Signaling Through CD40. Science 273:1862-1864, 1996.
Fisher KJ, Jooss K, Alston J, Yang Y, Haecker SE, High K, Pathak R, Raper SE, and Wilson JM. Recombinant Adeno-associated Virus for Muscle-Directed Gene Therapy. Nat Med 3:306-312, 1997.
Goldman MJ, Anderson GM, Stolzenberg ED, Kari UP, Zasloff M and Wilson JM. Human beta-Defensin I Is a Salt Sensitive Antibiotic in Lung That Is Disabled inCystic Fibrosis. Cell 88:553-560, 1997.
