Division of Medical Genetics

RESEARCH INTERESTS

James M. Wilson, M.D., Ph.D.

Dr. Wilson is interested in the study of inherited diseases and the development of effective therapies. One theme is the evaluation of cell biology relevant to organs affected in inherited diseases such as the lung in cystic fibrosis, the muscle in inherited muscular dystrophies and the liver in inborne errors of metabolism. Dr. Wilson's group uses animal models to evaluate the regenerative capacity of these organs as well as the existence of stem cells. In characterizing cystic fibrosis, Dr. Wilson's laboratory helped identify a defect in the innate immune system of the lung which contributes to the chronic airway respiratory infections characteristic of this disease. Molecules are present in the airway surface fluid which contribute to host defense; these have been characterized as a prelude to evaluating how they are deranged in CF. Therapeutic interventions primarily emphasize the use of somatic gene transfer to correct inherited defects. A number of studies utilize vectors based on DNA viruses such as recombinant adenovirus and adeno-associated virus. More recently, Dr. Wilson's group has exploited the biology of the lentiviral vector to achieve stable and long-term gene transfer in non-dividing cells.

Recent Publications

• Schnell MA, Zhang Y, Tazelaar J, Gao GP, Yu QC, Qian R, Chen S, Varnavski AN, LeClair C, Raper SE and Wilson JM. Activation of Innate Immunity in Nonhuman Primates Following Intraportal Administration of Adenoviral Vectors. Molecular Therapy, 3: 708-722, 2001.
• Varnavski AN, Zhang Y, Schnell M, Tazelaar J, Louboutin JP, Yu QC, Bagg A, Gao GP and Wilson JM. Preexisting Immunity to Adenovirus in Rhesus Monkeys Fails to Prevent Vector-Induced Toxicity. J Virol, 76:5711-5719, 2002.
• Auricchio A, Rivera VA, Clackson T, O'Conner E, Maguire AM, Tolentino MJ, Bennett J and Wilson JM. Pharmacological Regulation of Protein Expression from Adeno-Associated Viral Vectors in the Eye. Mol Thera, 6: 238-242, 2002.
• Wang X, Moser C, Louboutin JP, Lysenko ES, Weiner DJ, Weiser JN and Wilson JM. Toll Like Receptor 4 Mediates Innate Immune Responses to Haemophilus influenzae Infection in Mouse Lung. J Immun, 168:810-815, 2002.
• Gao GP, Alvira M, Wang L, Calcedo R, Johnston J and Wilson JM. Novel Adeno-Associated Viruses from Rhesus Monkeys as Vectors for Human Gene Therapy. Proc Natl Acad Sci USA, 99: 11854-11859, 2002.
• Gao GP, Alvira MR, Somanathan S, Lu Y, Vandenberghe LH, Rux JJ, Calcedo R, Sanmiguel J, Abbas Z and Wilson JM. Adeno-Associated Viruses Undergo Substantial Evolution in Primates During Natural Infections. Proc Natl Acad Sci USA, 100: 6081-6086, 2003.
• Medina MF, Kobinger GP, Rux J, Gasmi M, Looney DJ, Bates P and Wilson JM. Lentiviral Vectors Pseudotyped with Minimal Filovirus Envelopes Increased Gene Transfer in Murine Lung. Mol Thera, 8: 777-789, 2003.
• Kobinger GP, Louboutin JP, Barton ER, Sweeney HL and Wilson JM. Correction of the Dystrophic Phenotype by In Vivo Targeting of Muscle Progenitor Cells. Hum Gene Thera, 14: 1441-1449, 2003.