Weill-Cornell Overview 
    - Ronald G. Crystal, MD, Principal Investigator

The unifying theme of the Weill Cornell PEGT is the challenge of adapting the technology of ex vivo and in vivo gene transfer to treat and prevent disorders of heart, lung and blood. This challenge can be met by understanding the biology of gene transfer to cells and experimental animals and applying that understanding to the design of clinical studies. The Weill Cornell PEGT combines extensive gene therapy core facilities with 6 NIH funded Principal Investigators at Weill Cornell, Memorial-Sloan Kettering, and Evanston Northwestern, with overlapping interests and ongoing collaborations including 2 NIH Program Projects and 5 shared R01 grants. The proposed PEGT comprises 4 pre-clinical projects, 2 clinical projects, 8 cores, and a data management program. The projects include: (1) Genetic treatment of b-thalassemia by lentivirus-mediated transfer of a regulated human b-globin gene (M. Sadelain); (2) In vivo expansion, mobilization and recovery of bone marrow-derived stem cells by regional delivery of adenoviral vectors expressing cytokines (S. Rafii); (3) Development of a anti-Pseudomonas vaccine using dendritic cells modified to express CD40L and pulsed with Pseudomonas (R.Crystal); and (4) Prospective, placebo controlled, randomized assessment of adenovirus-mediated VEGF121 cDNA myocardial angiogenesis therapy as an adjunct to off-pump coronary artery bypass surgery (T. Rosengart). The supporting cores include: DNA vector; RNA vector; Stem cell; Analysis; Clinical Operations and Regulatory Affairs; Experimental Animal; Training and Education; Administration; and PEGT Data Management.

- Project 1 - Genetic Treatment of b-thalassemia by Lentivirus-Mediated transfer of a Regulated Human b-globin Gene. M. Sadelain

- Project 2 - In Vivo Expansion, Mobilization and Recovery of Bone Marrow-Derived Totipotent Stem Cells by Locoregional Delivery with Adenoviral Vectors Expressing Stem Cell Active Chemocytokines. S. Rafii

- Project 3 - Development of a T Cell-Independent Anti-Pseudomonas Vaccine Using Dendritic Cells Genetically Modified to Express CD40L and Pulsed with Pseudomonas. R. Crystal 

- Project 4 - Prospective, Placebo Controlled, Randomized Assessment of Adenovirus-Mediated VEGF121 cDNA Myocardial Angiogenesis Therapy as an Adjunct to Individuals with Diffuse Coronary Artery Disease Undergoing Off-Pump Coronary Artery Bypass Surgery. T. K. Rosengart 

- National Vector Production Cores: