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 the biology of gene transfer to cells and experimental animals and applying that understanding to the design of clinical studies. The Weill Cornel 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 - thalassemia by lentivirus-mediated transfer of a regulated human-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) Manipulation of hematopoietic and endothelial stem cell self-renewal and proliferation by adeno- and retroviral gene transfer (M. Moore); (4) Development of a anti-Pseudomonas vaccine using dendritic cells modified to express CD40L and pulsed with Pseudomonas (R. Crystal); (5) retroviral mediated transfer of the glucose-6-phosphate dehydrogenase gene into human hematopoietic progenitor cells for the treatment of patients with chronic non-spherocytic hemolytic anemia (L. Luzzato), 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.

National Institute of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
Research Project--Cooperative Agreements (U01)
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Special Emphasis Panel (ZHL1-CSR-C (S2))
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Weill Medical College of Cornell University
Schools of Medicine
New York
United States
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Chiuchiolo, Maria J; Crystal, Ronald G (2016) Gene Therapy for Alpha-1 Antitrypsin Deficiency Lung Disease. Ann Am Thorac Soc 13 Suppl 4:S352-69
Nolan, Daniel J; Ginsberg, Michael; Israely, Edo et al. (2013) Molecular signatures of tissue-specific microvascular endothelial cell heterogeneity in organ maintenance and regeneration. Dev Cell 26:204-19
Wang, Lan; Rosenberg, Jonathan B; De, Bishnu P et al. (2012) In vivo gene transfer strategies to achieve partial correction of von Willebrand disease. Hum Gene Ther 23:576-88
Krause, Anja; Whu, Wen Zhu; Xu, Yaqin et al. (2011) Protective anti-Pseudomonas aeruginosa humoral and cellular mucosal immunity by AdC7-mediated expression of the P. aeruginosa protein OprF. Vaccine 29:2131-9
Shmelkov, Sergey V; Hormigo, Adília; Jing, Deqiang et al. (2010) Slitrk5 deficiency impairs corticostriatal circuitry and leads to obsessive-compulsive-like behaviors in mice. Nat Med 16:598-602, 1p following 602
Rabbany, Sina Y; James, Daylon; Rafii, Shahin (2010) New dimensions in vascular engineering: opportunities for cancer biology. Tissue Eng Part A 16:2157-9
Wang, G; Qiu, J; Wang, R et al. (2010) Persistent expression of biologically active anti-HER2 antibody by AAVrh.10-mediated gene transfer. Cancer Gene Ther 17:559-70
Yamamoto, Masaya; James, Daylon; Li, Hui et al. (2010) Generation of stable co-cultures of vascular cells in a honeycomb alginate scaffold. Tissue Eng Part A 16:299-308
Watanabe, M; Boyer, J L; Crystal, R G (2010) AAVrh.10-mediated genetic delivery of bevacizumab to the pleura to provide local anti-VEGF to suppress growth of metastatic lung tumors. Gene Ther 17:1042-51
Butler, Jason M; Kobayashi, Hideki; Rafii, Shahin (2010) Instructive role of the vascular niche in promoting tumour growth and tissue repair by angiocrine factors. Nat Rev Cancer 10:138-46

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