Perspectives Page 1 Perspectives Gene Therapy for Cystic Fibrosis: Challenges and Future
Directions James M. Wilson Institute for Human Gene Therapy and Department ofMolecular and
Cellular Engineering, University of Pennsylvania, and the Wistar Institute, Philadelphia,
Pennsylvania 19104 The initial concept of human gene therapy was permanent correction of a
recessive disease by transplantation of ex vivo manipulated stem cells (1). An early and still
important model for this strategy is treatment of severe combined immunodeficiency due to …