CORE: Administrative Core Core Director: James M. Wilson, M.D., Ph.D. The administrative home for the management of this NIDDK Gene Therapy Program (GTP) CF Translational Research Consortium (GTP ? CFTRC) is the GTP of the Division of Transfusion Medicine in the Department of Pathology and Laboratory Medicine at UPenn which is headed by the PI of this grant, Dr. James M. Wilson. The Dean of the Perelman School of Medicine provides financial support to this program. Dr. Terence Flotte, the Dean of the School of Medicine as well as Provost & Executive Deputy Chancellor of the University of Massachusetts Medical School (UMMS), is the Associate Director of this grant. Ms. Kelly D. Reynolds, who is the Director of Finance and Operations for GTP, provides all administrative support for the activities of the Center. She and her staff will coordinate the financial and reporting requirements for all members of the GTP ? CFTRC. Dr. Maria P. Limberis at UPenn is the Center Coordinator and will facilitate engagement and collaboration of Center Members. A number of administrative vehicles are in place to assure the Center and its participating cores meet the objectives of the Program. Independent bi-weekly core management meetings are held to review activities of the Vector, Immunology, Animal Models, Cell Morphology and Ion Transport Cores. Internal and External Oversight Committees are in place to review the activities of the Center. This Center grant partially funds a Gene Therapy Seminar Series. Finally, Dr. Maria P. Limberis, together with Ms. Monique Molloy, will manage all aspects of the Pilot and Feasibility Program.

National Institute of Health (NIH)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Center Core Grants (P30)
Project #
Application #
Study Section
Special Emphasis Panel (ZDK1)
Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
University of Pennsylvania
United States
Zip Code
Svidritskiy, Egor; Korostelev, Andrei A (2018) Conformational Control of Translation Termination on the 70S Ribosome. Structure 26:821-828.e3
Svidritskiy, Egor; Korostelev, Andrei A (2018) Mechanism of Inhibition of Translation Termination by Blasticidin S. J Mol Biol 430:591-593
Gurda, Brittney L; De Guilhem De Lataillade, Adrien; Bell, Peter et al. (2016) Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII. Mol Ther 24:206-216
Svidritskiy, Egor; Madireddy, Rohini; Korostelev, Andrei A (2016) Structural Basis for Translation Termination on a Pseudouridylated Stop Codon. J Mol Biol 428:2228-36
Greig, Jenny A; Calcedo, Roberto; Grant, Rebecca L et al. (2016) Intramuscular administration of AAV overcomes pre-existing neutralizing antibodies in rhesus macaques. Vaccine 34:6323-6329
McClain, Lauren E; Davey, Marcus G; Zoltick, Phillip W et al. (2016) Vector serotype screening for use in ovine perinatal lung gene therapy. J Pediatr Surg 51:879-84
Calcedo, Roberto; Wilson, James M (2016) AAV Natural Infection Induces Broad Cross-Neutralizing Antibody Responses to Multiple AAV Serotypes in Chimpanzees. Hum Gene Ther Clin Dev 27:79-82
Bissig-Choisat, Beatrice; Wang, Lili; Legras, Xavier et al. (2015) Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model. Nat Commun 6:7339
Svidritskiy, Egor; Korostelev, Andrei A (2015) Ribosome Structure Reveals Preservation of Active Sites in the Presence of a P-Site Wobble Mismatch. Structure 23:2155-61
Wang, Lili; Bell, Peter; Somanathan, Suryanarayan et al. (2015) Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids. Mol Ther 23:1877-87

Showing the most recent 10 out of 231 publications