Curriculum Vitae Date : 15 February 2016 Guangping Gao, Ph.D. Home Address: 408 Yorkshire Road Rosemont, PA 19010-1119 610-527-4680 Office Address: 2000, Translational Research Laboratories 125S, 31st Street Philadelphia, PA 19104-3403 Tel: 215-898-2046 Cell: 484-686-7000 (Blackberry) Fax: 215-898-6588 Citizenship: U.S. Citizen Education: 1978-82 B.M. 1988-90 M.S. 1990-94 Ph.D. West China University of Medical Sciences Florida International University Florida International University (Biological Sciences) Postgraduate Training and Fellowship Appointments: 1988-89 1994-96 Fellowship, Biomedical Engineering World Health Organization/Florida International University, Miami Postdoctoral Fellow, Gene Therapy, Institute for Human Gene Therapy University of Pennsylvania, Philadelphia, PA Faculty /Staff Appointment: 1986-1988 Instructor in Biochemistry, Department of Biochemistry, College of Pharmaceutical Sciences, West China University of Medical Sciences, Chengdu, P.R. China 1996-1998 Assistant Director, Human Applications Laboratory, Institute for Human Gene Therapy University of Pennsylvania, Philadelphia, PA 1998-2002 Associate Director, Institute for Human Gene Therapy University of Pennsylvania, Philadelphia, PA 2003-present Director, Vector Program, Gene Therapy Program, Department of Medicine Division of Medical Genetics, School of Medicine University of Pennsylvania, Philadelphia, PA Other Appointments 2006-present Deputy Director, Vaccine Research Institute Joint Research Program of University of Pennsylvania and Sun Yat-Sen University Guangzhou, China Chair, Manufacturing Committee Guangping Gao, M.D., Ph.D. Page 2 International AAV Vector Reference Standard Working Group Co-chairs for AAV vector sessions at annual meetings for American Society of Gene Therapy Abstract review and selection committees for ASGT Annual Meetings Scientific review board of “Human Gene Therapy” Ad hoc reviewer for Current Gene Therapy Gene Therapy Journal of Virology Molecular Therapy Nature Medicine Nature Biotechnology And other gene therapy and virology journals Invited for grant reviews by the following study sections: NIH/NIAID, Integrated Clinical/Pre-Clinical HIV Therapeutics NIH/NIDDK, Centers of Excellence in Hematology Research Grants Council of Hong Kong, Infectious Diseases Association Française contre les Myopathies Membership in Honorary Societies 1989 Phi Kappa Phi Memberships in Professional and Scientific Societies: American Society of Human Genetics American Society of Gene Therapy American Association for the Advancement of Science Lectures by Invitation and Chaired sessions at International conferences : April 1, 1994 Baylor Medical College, Houston, TX Sept 7, 1996 The 3rd Annual Retreat of the Institute for Human Gene Therapy, Absecon, NJ June 3, 1997 Cystic Fibrosis Foundation Conference, Gene Therapy Session, Williamsburg, VA Nov 8, 1998 The 5th Annual Retreat of the Institute for Human Gene Therapy, Absecon, NJ June 9, 2002 American Society of Gene Therapy Annual Meeting, Boston, MA August 28, 2002 IX Parvovirus Workshop, Bologna, Italy June 5, 2003 American Society of Gene Therapy Annual Meeting, Washington, DC June 6, 2003 American Society of Gene Therapy Annual Meeting, Washington, DC March 4, 2004 University of North Carolina at Chapel Hill June 2, 2004 American Society of Gene Therapy Annual Meeting, Minneapolis, ME Guangping Gao, M.D., Ph.D. Page 3 June 4, 2004 American Society of Gene Therapy Annual Meeting, Minneapolis, ME Sept. 8, 2004 X Parvovirus Workshop, St. Petersburg, Florida Sept. 30, 2004 NHF 7th Workshop on novel technologies and gene transfer for hemophilia B, Philadelphia, PA Oct. 18, 2004 Symposium of Gene Therapy, Taipei, Taiwan Dec. 3, 2004 University of Pittsburgh, Pittsburgh, PA May 8, 2005 Gene Therapy Symposium, The Institute of Biomedical Sciences, Academia Sinica Taipei, Taiwan June 1, 2005 American Society of Gene Therapy Annual meeting, St. Louis, USA. June 3, 2005 American Society of Gene Therapy Annual meeting, St. Louis, USA. August 8, 2005 International conference for novel Biological Product Development, Mclean, VA, U.S.A. March 31, 2006 NHF 8th Workshop on novel technologies and gene transfer for hemophilia B, Philadelphia, PA May 30, 2006 American Society of Gene Therapy Annual meeting, Baltimore, MD June 24, 2006 AAV gene vector manufacturing workshop, Organized by the Monegasque Association against Muscular Dystrophy, Duchenne Parent Project France and Parent Project UK Monte Carlo, Monaco August 30, 2006 XI Parvovirus Workshop, Les Diablerets, Switzerland June 3, 2007 American Society of Gene Therapy Annual Meeting, Seattle, WA July 5th, 2007 Chinese Institute of Medical Sciences, Beijing, China July 6th, 2007 Vector Gene Transfer Company, Beijing, China Bibliography: Chapters in Books and Reviews: 1. Gao, G.P., Wivel N., and James M. Wilson. Production of Recombinant AAV, Viral Vectors for Gene Therapy. In Advances in Virus Research. Edited by J.C. Glorioso. Academic Press, New York, 55:529-543, 2000 2. Wivel N., Gao, G.P., and James M. Wilson. Adenoviral Vectors. The Development of Human Gene Therapy. Edited by Theodore Friedman, Cold Spring Harbor Laboratory Press, New York, pp.87-110, 1999 3. Gao, G.P., Vandenberghe, L.H. and Wilson J.M. New Serotypes of AAV Vectors. Current Gene Therapy, 2005, (3):285-97 4. Gao, G.P., Vandenberghe, L.H. and Wilson J.M. Identification of Novel AAV Serotypes for Use as Vectors. GENE THERAPY IN THE BRAIN, Edited by Michael G. Kaplitt and Matthew During, 2006. Research Publications, Peer Reviewed: 1. Susan L. Pichla_Gollon, Mark Drinker, Xiangyang Zhou, Feng Xue, John J. Rux, Guangping Gao, James M. Wilson, Guangping Gao, M.D., Ph.D. Page 4 Hildegund C.J. Ertl, Roger M. Burnett and Jeffrey M. Bergelson. Structure-Based Identification of a Major Neutralizing Site in an Adenovirus Hexon. J. Virol. In press. 2. Luk H. Vandenberghe, Lili Wang, Suryanarayam Somanathan, Yan Zhi, Joanita Figueredo, Roberto Calcedo, Julio Sanmiguel, Ravi A. Desai, Christopher S. Chen, Julie Johnston, Rebecca L. Grant, Guangping Gao, and James M. Wilson. Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med, 12:967-971, 2006. 3. Zhi Y, Figeuredo J, Kobinger G, Hagan H, Calcedo R, Miller J, Gao G and Wilson JM. Efficacy of a SARS vaccine based on a non-human primate adenovirus in the presence of immunity against human adenovirus. Hum Gene Ther, 17:500-506, 2006 4. Gao G, Yu L, Johnston J, Calcedo R, Grant R and Wilson JM. High level transgene expression in nonhuman primate liver with novel AAV serotypes containing self-complementary genomes. J Virol, 80:6192-6194, 2006. 5. Kelkar S, De B, Gao G, Wilson JM, Crystal RG and Leopold PL. A common mechanism for cytoplasmic dynein-dependent microtubule binding shared among adeno-associated virus and adenovirus serotypes. J Virol, 80:7781-7785, 2006. 6. Gao GP, You Lu, Roberto Caceldo, Grant Rebecca and James Wilson: Biology of novel AAV serotype vector mediated liver gene transfer in NHPs. Molecular Therapy, 13:77-87, 2006. 7. Kitajima K, Miller GC, Gao G, Marchadier DHL, Rader D and Wilson JM. Complete prevention of atherosclerosis in apoE deficient mice by hepatic human apoE gene transfer with adeno-associated virus serotypes 7 and 8. Arterioscler Thromb Vasc Biol, 26:1852-1857, 2006. 8. Kobinger GP, Feldmann H, Zhi Y, Schumer G, Gao GP, Feldmann F, Jones S and Wilson JM. Simian Adenovirus Vaccines Protects Against Zaire Ebola Virus, J Virol, 346:394-401, 2006. 9. De BD, Heguy A, Hackett NR, Lee J, Pierre L, Gao G, Wilson JM and Crystal RG. High levels of persistent expression of α1-antitrypsin mediated by the non-human primate serotype rh.10 adeno- associated virus despite pre-existing immunity to common human adeno-associated viruses. Mol Ther, Mol Ther, 13:67-76, 2006 10. Tatsis N, Tesma L, Robinson E, Giles-Davis W, McCoy K, Gao G, Wilson JM and Ertl HCJ. Chimpanzee-origin adenovirus vectors as vaccine carriers. Gene Ther, 13:421-429, 2006 11. Moscioni AD, Morizono H, McCarter RJ, Stern A, Cabrera-Luque J, Hoang A, Sanmiguel J, Wu D, Bell P, Gao G, Raper SE, Batshaw ML and Wilson JM. Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with novel adeno-associated viral vectors. Mol Ther, 14:24-33, 2006. 12. Zhi Y, Kobinger GP, Jordan H, Suchma K, Shen H, Gao GP, Boyer JL, Crystal RG and Wilson JM. Identification of Murine CD8 T Cell Epitopes in Codon Optimized SARS-Associated Coronavirus Spike Protein. Virology, 335:34-45, 2005. 13. Bell P, Limberis M, Gao GP, Wu D, Bove MS, Sanmiguel J and Wilson JM. An Optimized Protocol for Detection of E. coli β- galactosidase in Lung Tissue following Gene Transfer. Histochem Cell Biol, 124:77-85, 2005. 14. Bell P, Wang L, Lebherz C, Flieder DB, Bove MS, Wu D, Gao GP, Wilson JM and Wivel NA. No Evidence for Tumorigenesis of AAV Vectors in a Large Scale Study in Mice. Mol Ther, 12:299 306, 2005. 15. Lucy Deriy, Haruko Ogawa, Guangping Gao, and Uri Galili. In vivo targeting of vaccinating tumor cells to antigenpresenting cells by a gene therapy method with adenovirus containing the 1,3 galactosyltransferase gene. Cancer Gene Therapy. 2005, 12:528-539. 16. Jennings K, Miyamae T, Traister R, Marinov A, Katakura S, Sowders D, Trapnell B, Wilson JM, Gao GP and Hirsch R. Proteasome Inhibition Enhances AAV-Mediated Transgene Expression in cytes in Vitro and in Vivo. Mol Ther, 11:600-607, 2005. Guangping Gao, M.D., Ph.D. Page 5 17. Li Z, Qiao H, Lebherz, Choi SR, Zhou X, Gao G, Kung HF, Rader DJ, Wilson JM, Glickson JD and Zhou R. Creatine Kinase, a Magnetic Resonance-Detectable Marker Gene for Quantification of Liver-Directed Gene Transfer. Hum Gene Ther, 16:1429-1438, 2005. 18. Rivera VM, Gao GP, Grant RL, Schnell MA, Zoltick PJ, Rozamus LW, Clackson T and Wilson JM. Long-Term Pharmacologically Regulated Expression of Erythropoietin in Primates following AAV-Mediated Gene Transfer. Blood, 105:1424-30, 2005 19. Varnavski AN, Calcedo R, Bove M, Gao GP and Wilson JM. Evaluation of Toxicity from High-Dose Systemic Administration of Recombinant Adenovirus Vector in Vector-Naïve and Pre-immunized Mice. Gene Thera, 12:427-436, 2005 20. Katakura S, Jennings K, Watanabe S, Adachi E, Thornton S, Gao GP, Wilson JM, Burstein H, Trapnell B and Hirsch R. Recombinant Adeno-Associated Virus Preferentially Transduces Human, Compared to Mouse, Synovium: Implications for Arthritis Therapy. Mod Rheumatol, 14: 18-24, 2004. 21. Hogan RJ, Gao GP, Rowe T, Bell P, Flieder D, Paragas J, Kobinger GP, Wivel NA, Crystal RG, Boyer J, Feldmann H, Voss TG and Wilson JM. Resolution of Primary SARS-Associated Coronavirus Infection Requires Stat-1. J Virol,, 2004. 22. Rowe T, Gao GP, Hogan RJ, Crystal RG, Voss TG, Grant RL, Bell P, Kobinger GP, Wivel NA and Wilson JM. Macaque Model for SARS. J Virol,, 2004. 23. Roy S, Gao GP, Clawson D, Farina SF and Wilson JM. Complete Nucleotide Sequences and Genome Organization of Four Chimpanzee Adenoviruses. Virology, 324: 361-372, 2004. 24. Gao GP*, Roy S*., Lu Y, Zhou X, Lock M, Calcedo R and Wilson JM. Characterization of a Family of Chimpanzee Adenoviruses and Development of Molecular Clones for Gene Transfer Vectors. Hum Gene Thera, 15: 519-530, 2004. *Equal contribution. 25. Reyes-Sandoval A, Fitzgerald JC, Grant R, Roy S, Xiang ZQ, Li Y, Gao GP, Wilson JM and Ertl HCJ. HIV-1 Specific Immune Responses in Primates upon Sequential Immunization with Adenoviral Vaccine Carriers of Human and Simian Serotypes. J Virol, 78: 7392-7399, 2004. 26. Gao GP, Luk H. Vandenberghe, Mauricio R. Alvira, You Lu, Roberto Calcedo, Xiangyang Zhou and James M. Wilson. Clades of Adeno-Associated Viruses are Widely Disseminated in Human Tissues. J Virol, 78: 6381-6388, 2004 27. Ziegler RJ, Lonning SM, Armentano D, Li C, Souza DW, Cherry M, Ford C, Barbon CM, Desnick RJ, Gao GP, Wilson JM, Peluso R, Godwin S, Carter BJ, Gregory RJ, Wadsworth SC and Cheng SH. AAV2 Vector Harboring a Liver-Restricted P -galactosidase A and the Induction of Immune Tolerance in Fabry Mice. Mol Thera, 9: 231-240, 2004 28. Gao GP, Lebherz C, Weiner DJ, Grant R, Calcedo R, Bagg A, Zhang Y and Wilson JM. Erythropoietin Gene Therapy Leads to Autoimmune Anemia in Macaques. Blood, 103: 3300-3302, 2004. 29. Lebherz C, Gao GP, Louboutin JP, Millar J, Rader D and Wilson JM. Gene Therapy with Novel Adeno-Associated Virus Vectors Substantially Diminishes Atherosclerosis in a Murine Model of Familial Hypercholesterolemia. J of Gene Med, 6: 663-672, 2004 30. Pinto AR, Fitzgerald J, Gao GP, Wilson JM and Ertl HCJ. Induction of CD8+ T Cells to an HIV-1 Antigen upon Oral Immunization of Mice with a Simian E1-Deleted Adenoviral Vector. J Immunol, 171: 6774-6779, 2003 31. Xiang ZQ, Li Y, Gao GP, Wilson JM and Ertl HJC. Mucosally Delivered E1-Deleted Adenoviral Vaccine Carriers Induce Transgene Product-Specific Antibody Responses in Neonatal Mice. J Immunol, 171:4287-4293, 2003. 32. Raper SE, Chirmule N, Lee FS, Bagg A, Gao GP, Heidenreich R, Wilson JM and Batshaw ML. Fatal Systemic Inflammatory Response Syndrome in an Ornithine Transcarbamylase Deficient Patient following Adenoviral Gene Transfer. Mol Gen and Metab, 80: 148-158, 2003. Guangping Gao, M.D., Ph.D. Page 6 33. Sarkar R, Tetreault R, Gao GP, Wang L, Bell P, Chandler R, Wilson JM and Kazazian HH. Total Correction of Hemophilia A Mice with Canine FVIII Using an AAV8 Serotype. Blood, epub ahead of print, Oct 9, accession number 14551134, 2003. 34. Xiang ZQ, Gao GP, Reyes-Sandoval A, Li Y, Wilson JM and Ertl HC. Oral Vaccination of Mice with Adenoviral Vectors is not Impaired by Preexisting Immunity to the Vaccine Carrier. J Virol, 77: 10780-10789, 2003. 35. Pinto AR, Fitzgerald JC, Giles-Davis W, Gao GP, Wilson JM and Ertl HCJ. Induction of CD8+ T Cells to an HIV-1 Antigen through a Prime Boost Regimen with Heterologous E1-Deleted Adenoviral Vaccine Carriers. J Immunol, 171: 6774-6779, 2003. 36. Vinner L, Wee EGT, Patel S, Corbet S, Gao GP, Nielsen C, Wilson JM, Ertl HCJ, Hanke T and Fomsgaard A. Immunogenicity in Mamu-A*01 Rhesus Macaques of a CCR5-Tropic Human Immunodeficiency Virus Type 1 Envelope from the Primary Isolate (Bx08) after Synthetic DNA Prime and Recombinant Adenovirus 5 Boost. J Gen Virol, 84: 203-213, 2003. 37. Gao, G.P., Alvira, M.R., Somanathan, S., Lu, Y., Sanmiguel, J., Abbas, Z., Johnston, J., and Wilson, J.M. Adenoassociated viruses undergo substantial evolution in primates during natural infections. PNAS, 100:6081-6086, 2003. 38. Gao GP, Zhou X, Alvira MR, Tran P, Marsh J, Lynd K, Xiao W and Wilson JM. High Throughput Creation of Recombinant Adenovirus Vectors by Direct Cloning, Green White Selection and I-Sce I Mediated Rescue of Circular Adenovirus Plasmids in 293 Cells. Gene Therapy, 10: 1926-1930, 2003. 39. Xiang ZQ, Gao GP, Li Y, Wilson JM and Ertl HCJ. T Helper Cell- Independent Antibody Responses to the Transgene Product of an E1-Deleted Adenoviral Vaccine Require NK1.1 T Cells. Virology, 305: 397-405, 2003 40. Fitzgerald J, Gao GP, Reyes-Sandoval A, Pavlakis GN, Xiang ZQ, Wlazlo AP, Giles-Davis W, Wilson JM and Ertl HCJ. A Simian Replication-Defective Adenoviral Recombinant Vaccine to HIV-1 Gag. J Immunol, 170: 1416-1422, 2003 41. Johnston J, Tazelaar J, Rivera VM, Clackson T, Gao GP and Wilson JM. Regulated Expression of Erythropoietin from an AAV Vector Safely Improves the Anemia of β-thalassemia in a Mouse Model. Mol Thera, in press. 42. Varnavski AN, Schlienger K, Bergelson JM, Gao GP and Wilson JM. Efficient Transduction of Human Monocyte-Derived Dendritic Cells by Chimpanzee-Derived Adenoviral Vector. Hum Gene Thera, 14:533-544, 2003 43. Green AP, Huang JJ, Scott MO, Beaupre I, Meyers A, Gao GP and Wilson JM. A New Scaleable Method for the Purification of Recombinant Adenovirus Vectors. Hum Gene Thera, 13:1921-1934, 2002. 44. Gao, G.P., Alvira, M., Wang, L., Calcedo, R., and Wilson, J.M. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA, 99: 11854-11859, 2002. 45. Auricchio, A., O’Connor, E., Weiner, D., Gao, G.P., Hildinger, M., Wang, L., and Wilson, J.M. Non-invasive gene transfer to lung for systemic delivery of therapeutic proteins. J Clin Invest, in press, 2002. 46. Auricchio, A., Gao, G.P., Yu, Q.C., Raper, S., Rivera V., and Wilson, J.M. Constitutive and regulated expression of processed insulin following In Vivo Hepatic Gene Transfer. Gene Thera, 9:962-970, 2002. 47. Derily L., Chen ZC, Gao GP and Galili U. Expression of -gal epitopes on HeLa cells transduced with adenovirus containing 1,3 galactosyltransferase cDNA. Glycobiology, 2002, 12:135-144. 48. Cohen, C., Xiang, Z.Q., Gao, G.P., Ertl, H.C.J., Wilson, J.M., and Bergelson, J.M. Chimapanzee adenovirus 68 adapted as a gene delivery vector interacts with the coxsackievirus and adenovirus receptor (CAR). J Gen Virol, 83:151-155, 2002. 49. Gao, G.P., Lu, F.M., Sanmiguel, J.C., Tran, PT., Abbas, Z., Lynd, K.S., Marsh, J., Spinner, N.B., and Wilson, J.M. Rep/Cap gene amplification and high yield production of AAV in an A549-Cell line expressing Rep/Cap. Mol Thera, 5:644-649, 2002. Guangping Gao, M.D., Ph.D. Page 7 50. Varnavski, A.N., Zhang, Y., Schnell, M., Tazelaar, J., Louboutin, J.P., Yu Q.C., Bagg, A., Gao, G.P., and Wilson, J.M. Preexisting immunity to Adenovirus in rhesus monkeys fails to prevent vector-induced toxicity. J Virol, 76:5711-5719, 2002. 51. Xiang, Z.Q., Gao, G.P., Reyes-Synoval, A., Cohen, C.J., Li Y., Wilson, J.and Ertl, H.C.J. Novel, chimpanzee serotype 68based adenoviral vaccine carrier for induction of antibodies to a transgene product. J Virol, 76:2667-2675, 2002. 52. Auricchio, A., Hildinger, M., O’Connor, E., Gao, G.P. and Wilson J.M. Isolation of highly infectious and pure AAV2 vectors with a single-step gravity-flow column. Hum Gene Thera, 12:71-76, 2001. 53. Cordier, L., Gao, G.P., Hack, A.A., McNally, E.M., Wilson, J.M., Chirmule, N., and Sweeney, H.L. Muscle-specific promoters may be necessary for AAV-mediated gene transfer in the treatment of muscular dystrophies. Hum Gene Thera, 12: 205-215,2001. 54. Cordier, L, Gao, G.P., Wilson, J.M., Chirmule, N., and Sweeney, L.H. Use of muscle-specific promoter may be necessaryfor AAV-mediated gene transfer in the treatment of muscle dystrophies. Hum Gene Thera, 12:205-215, 2001. 55. Dejneka, N.S., Auricchio, A., Maguire, A.M., Ye, X., Gao, G.P., Wilson, J.M. and Bennett, J. Pharmacologically-regulated gene expression in the retina following transduction with viral vectors. Gene Thera, 8:442-446, 2001 56. Eck, S.L., Alavi, J.B., Judy, K., Phillips, P., Alavi, A., Hackney, D., Cross, P., Hughes, J., Gao, G.P., Wilson, J.M., and Propert, K. Treatment of recurrent or progressive malignant glioma with a recombinant adenovirus expressing human interferon-beta (H5.010CMVhIFN- ): A phase I trial. Hum Gene Thera, 12:97-113, 2001. 57. *Gao, G.P., *Farina, S.F., Xiang, Z.Q., Rux, J.J., Burnett, R.M., Alvira, M.R., Marsh, J., Ertl, H.C.J., and Wilson, J.M. A replication defective vector based on a chimpanzee adenovirus. J Virol, 75:11606-11613, 2001. . *Equal contribution. 58. *Gao, G.P., *Tao, N., *Parr, M., Johnston, J., Baradet T., Wilson, J.M., Barsoum, J., and Fawell, S.E. Sequestration of adenoviral vector by Kupffer cells lads to a non-linear dose response of transduction in liver. Mol Therapy, 3:28-35, 2001.*Equal contribution 59. Hildinger, M., Auricchio, A., Gao, G.P., Wang, L., Chirmule, N.and Wilson, J.M. Hybrid vectors based on adeno- associated virus serotypes 2 and 5 for muscle-directed gene transfer. J Virol, 75:6199-6203, 2001. 60. Odaka, M., Sterman, D.H., Wiewordt, R., Zhang, Y., Kiefer, Amin K., Gao, G.P., Wilson, J.M., Barsoum J., Kaiser, L. and Albelda, S.M. Eradication of intraperitoneal and distant tumor by adenovirus mediated interferon beta gene transfer due to induction of systematic immunity. Cancer Research, 61:6201-6212, 2001 61. Raper, S.E., Yukoff, M., Chirmule, N., Gao., G.P., Nunes, F., Haskal, Z.J., Furth, E.E., Propert, K.J., Robinson, M.B., Magosin, S., Simoes, H., Speicher, L., Hughes, J., Tazelaar, J., Wivel, N.A., Wilson, J.M. and Batshaw, M.L. A pilot study of in vivo liver-directed gene transfer with an adenovirus vector in partial ornithine transcarbamylase deficiency. Hum Gene Thera, 13:163-175, 2001. 62. Schnell, M.A., Zhang, Y., Tazelaar, J., Gao, G.P., Yu, Q.C., Qian, R., Chen, S., Varnavski, A.N., LeClair, C., Raper, S.E., and Wilson, J.M. Activation of innate immunity in nonhuman primates following intraportal administration of adenoviral vectors. Mol Therapy, 3:708-722, 2001. 63. Zhang, Y., Schnell, M.A., Tazelaar, J., Gao., G.P., Yu, Q.C., Qian, R., Chen, S., Varnavski, A., Le Clair C., Raper S.E., and Wilson, J.M. Activation of innate immunity in nonhuman primates following intraportal administration of adenovirus vectors. Mol Therapy, (in press), 2001. 64. Zhang, Y., Chirmule, N., Gao., G.P., Qian, R., Croyle, MA., Joshi, B.,Tazelaar, J., and Wilson, J.M. Acute cytokine response to systematic adenoviral vectors in mice is mediated by dendritic cells and macrophages. Mol Therapy, 3:697-707, 2001. 65. Zoltick, P.W., Chirmule N., Schnell M.A., Gao, G.P., Hughes, J.V. and Wilson, J.M. Biology of E1-deleted adenovirus vectors in nonhuman primate muscle. J Virol, 75:5222-5229, 2001. Guangping Gao, M.D., Ph.D. Page 8 66. Cordier, L., Hack, A.A., Scott, M.O., Barton-Davis, E.R., Gao, G.P., Wilson, J.M., McNally, E.M., and Sweeney, H.L. Rescue of skeletal muscles of Gamma-sarcoglycan deficient mice with AAV-mediated gene transfer. Mol Therapy, 1: 119129. 2000 67. Chen, J.C., Rader, D.J., Tazelaar, J., Kawashiri, M., Gao, G.P., and Wilson, J.M. Prolonged correction of hyperlipidemia in mice with familial hypercholesterolemia using an adeno-associated viral vector expressing VLDL receptor. Mol Therapy, 2:256-261, 2000 68. Gao, G.P., Qu, G., Burnham, M.S., Huang, J., Chirmule N., Joshi., B., Yu, Q.C., Marsh, J .M, Conceicao C. A. and Wilson, J.M. Purification of recombinant adeno-associated virus vectors by column chromatography and its performance in vivo. Hum Gene Thera, 11:2079-2091, 2000. 69. Gao, G.P., Engdahl, R.K. and Wilson, J.M. A cell line for high-yield production of E1-deleted adenovirus vectors without the emergence of replication-competent virus. Hum Gene Thera, 11:213-219, 2000. 70. Sarkar, R., Gao, G.P., Chirmule R., Tazzelaar, J., and Kazazian, H. Jr. Partial correction of murine hemophilia A with Neoantigenic murine factor V III. Hum Gene Thera , 11:881-894, 2000. 71. Watanabe, S., Imagawa, T., Boivin, G.P., Gao, G.P., Wilson, J.M., And Hirsch, R. Adeno-associated virus mediates longterm gene transfer and delivery of chondroprotective IL-4 to murine synovium. Mol Therapy. 2:147-152,2000 72. Zhang, Y., Chirmule, N., Gao, G.P., and Wilson, J.M. CD40 ligand dependent activation of cytotoxic T lymphocytes by AAV vectors in vivo: Role of immature dendratic cells. J Virol, 74:8003-8010, 2000 73. Bennett J., Maguire A.M., Cideciyan A.V., Schnell M., Glover E., Anand V., Aleman T.S., Chirmule N., Gupta A.R., Huang Y., Gao G.P., Nyberg W.C., Tazelaar J., Hughes J., Wilson J.M., and Jacobson S.G. Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina. Proc Natl Acad Sci USA, 96:9920-9925, 1999. 74. Chirmule, N., Truneh, A., Haecker, S., Tazelaar, J., Gao, G.P., Raper, S., Hughes, J.V., Wilson, J.M. Repeated administration of adenoviral vectors in lungs of human CD4 transgenic mice treated with a non-depleting CD4 antibody. J. Immunol, 163:448-455, 1999. 75. Greelish JP., Su L.T., Lankford E.B., Burkman J.M., Chen H., Konig S.K., Mercier I.M., Desjardins P.R., Mitchell M.A., Zheng X.G., Leverovich J., Gao G.P., Balice-Gordon R.J., Wilson J.M. and Stedman H. Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector. Nat Med, 5:439-443, 1999. 76. Grifman M, Chen N.N., Gao, G.P., Cathomen T., Wilson J.M. and Weitzman M. Overexpression of cyclin A inhibits augmentation of recombinant adeno-associated virus transduction by the adenovirus E4orf6 protein. J Virol, 73: 1001010019, 1999. 77. Lanuti, M., Gao, G.P., Force, S.D., Chang, M.Y., E1 Kouri, C., Amin, K., Hughes, J.V., Wilson, J.M., Kaiser, L.R., Albelda, S.M. Evaluation of an E1/E4-deleted adenovirus expressing the herpes simplex thymidine kinase suicide gene in cancer gene therapy. Hum Gene Thera, 10:463-475, 1999. 78. Xiao, W.D., Chirmule, N., Berta, S.C., McCullough, B., Gao, G.P., Wilson, J.M. Gene Therapy Vectors Based on Adenoassociated Virus Type I. J Virol, 73:3994-4003, 1999. 79. Ye X.H., Rivera, V.M., Zoltick, P., Cerasoli F., Schnell, M.A., Gao, G.P., Hughes, J.V., Gilman, M., Wilson, J.M. Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science, 283:88-91, 1999. 80. Chirmule, N., Hughes, J.V., Gao, G.P., Raper, E.S., and Wilson, J.M. The role of E4 in eliciting CD4 T-cell and B-cell responses to adenoviral vectors delivered to murine and nonhuman primate lungs. J Virol, 72:6138-6145, 1998. Guangping Gao, M.D., Ph.D. Page 9 81. Gao, G.P., Qu, G., Faust, Lynn Z., Engdahl, Ryan K., Xiao, W.D., Hughes, J.V., Zoltick, P.W., and Wilson, J.M. High-titer adeno-associated viral vectors from a rep/cap cell line and hybrid shuttle virus. Hum Gene Thera, 9:2353-2362, 1998. 82. Raper, S.E., Haskal, Z.J., Ye, X.H., Pugh, C., Furth, E.E., Gao, G.P., and Wilson, J.M. Selective gene transfer into the liver of non-human primates with E1-deleted, E2A- defective or E1-E4 deleted recombinant adenoviruses: a preclinical toxicology study. Hum Gene Thera, 9:671-679, 1998. 83. Ye X., Gao G-P, Pabin C, Raper S.E. and Wilson J.M. Evaluating the potential of germ line transmission after intravenous administration of recombinant adenovirus in the C3H mouse. Hum Gene Thera, 9:2135-2142, 1998. 84. Fisher, K.J., Gao, G.P., Weitzman, M.D., Dematteo, R., Burda,J.F., and Wilson, J.M. Trandsduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol, 70:520-532, 1996. 85. Gao, G.P., Yang, Y.P., and Wilson J.M. Biology of adenoviral vectors deleted of E1 and E4 for liver-directed gene therapy. J. Virol, 70:8934-8943, 1996. 86. Gao, G.P. and Herrera R. Enrichment of middle repetitive element Bm-1 transcripts in fractions of the silkmoth, Bombyx mori. Genetica, 97:173-182, 1996. translationally active RNA 87. Kaul, R*., Gao, G.P., Matalon, R., Aloya, M., Su, Q., Jin, M., Johnson, A.B., Schutgens, R.B.H., and Clarke, J.T.R. Identification and expression of eight novel mutations among non-Jewish patients with Canavan disease. Am J Hum Genet 59:95-102, 1996. 88. Kaul, R*., Gao, G.P., Michals, K., Whelan, D., Levin, S.,and Matalon, R. (1995) Novel cys152 -> arg missense mutation in Arab patients with Canavan disease. Hum Mutat, 5:269-271, 1995. 89. Matalon, R., Kaul, R*., Gao, G.P., Michals, K., Gray, R.G.F., Bennett-Briton, S., Norman, A., Smith, M., and Jakobs, Prenatal diagnosis for Canavan disease: the use of DNA markers. J. Inhert Metabol Dis, 16:215-217, 1995. 90. Kaul, R*., Balamurugan, K., Gao, G.P., and Matalon, R. Canavan disease: genomic organization of human ASPA to 17p13ter; conservation of the ASP gene during evolution. Genomics, 2:364-370, 1994. 91. Kaul, R*., Gao, G.P., Balamurugan, K., and Matalon, R. Canavan Disease: molecular basis of spartoacylase deficiency. J Inhert Metabol Dis,. 17:295-297, 1994. 92. Kaul, R*., Gao, G.P., Aloya, M., Balamurugan, K., Petrosky, A., Michals, K., and Matalon, R*. Canavan Disease: mutations among Jewish and Non-Jewish Patients. Am J Hum Genet, 55:34-4, 1994. 93. Kaul, R*., Gao, G.P., Balamurugan, K., and Matalon, R. Cloning of the human aspartoacylase cDNA and a common missense mutation in Canavan Disease. Nature Genet, 5:118-123, 1993. *Ph.D. dissertation advisor and the publications generated from my dissertation project 94. von Sternberg, R.M., Novick, G.E., Gao, G.P., and Herrera, R.J. Genome canalization: the coevolution of transposable and Interspersed repetitive elements with single copy DNA Genetica, 86:215-246, 1992. 95. Yang, J.Y., Gao, G.P., Peng, X.F., Zeng, R., and Zhu, X.F. Studies on the new technique of preparing pancreatin from swine pancreas in accordance with the standard of the three enzymes in the Chinese Pharmacopeia, (1986). West China J. Pharmaceutical Sci., 3:15-18, 1988. 96. Yang, JY., Gao, G.P., Peng, X.F., Zeng, R., and Zhu, X.F. The relation among the activities of three enzymes in the process of preparing pancreatin from swine pancreas. West China J. Pharmaceutical Sci, 2:198-202, 1988. Patents: 1. Gao.G.P, Wilson,J.M, and Ertl. Method for rapid screening of bacterial transformants and novel simian adenovirus proteins. Guangping Gao, M.D., Ph.D. Page 10 2. Gao, G.P., Wilson, J.M. and Zhou, X. Y. Polyvalent viral vectors and system for production thereof. 3. Vandenberghe, L., Gao, G.P., and Wilson, J.M. Method of increasing the function of an AAV vector. 4. Wilson,J.M, Gao.G.P, Vandenberghe, L.H and Mauricio, R. A. AAV Clades, Sequences, vectors contained same, and uses Thereof 5. Gao, G.P and Wilson J.M. Method for direct rescue and amplification of integrated viruses from cellular DNA of tissues. 6. Gao, G.P. Wilson, J.M, and Alvira MR. Method of detecting and/or identifying AAV Sequences and isolating novel sequences identified thereby. 7. Gao, G.P. Wilson, J.M, and Alvira MR. Novel AAV Serotype 8 Sequences, and vectors containing same, and uses therefore 8. Gao, G.P. Wilson, J.M, and Alvira MR. Novel AAV Serotype 9 Sequences and uses therefore 9. Gao, G.P. and Wilson, J.M. Methods for efficient rescue and high throughput creation of adenovirus vectors. 10. Wilson, J.M., Gao, G.P. , and Roy, S. Chimpanzee adenovirus nucleic acid and amino acid sequences, vector construction containing same, and methods of use. 11. Gao, G.P. , Wilson, J.M and Alvira, M.R., Compositions and methods for production of recombinant viruses and uses therefore. 12. Zoltick.P, Gao, G.P. and Wilson.J.M. Regulatable gene expression system. 13. Gao, G.P. and Wilson, J.M. Compositions and methods for increasing packaging and yields of recombinant adenoviruses using multiple packaging signals. 14. Gao G.P. and Wilson, J.M. Cell lines and constructs useful in production of E1-Deleted adenovirus in absence of replication competent adenovirus. 15. Gao, G.P. and Wilson, J.M. Compositions ands Methods for helper-free production of recombinant adeno-associate viruses. 16. Gao, G.P. and Wilson, J.M., and Methods and cells lines for production of recombinant adeno-associated viruses. 17. Gao, G.P. and Wilson, J.M., and Novel adenovirus gene therapy Vehicle and cell lines. 18. Wilson, J.M., Fisher, K., and Gao, G.P. Recombinant adenovirus and adeno-associated virus. 19. Wilson, J.M., and Gao, G.P. Method for improved production of recombinant adenovirus vectors. 20. Matalon, R., Kaul, R., Gao, G.P., Balamurugan, K., Michals-Matalon, K. Aspartoacylase gene, protein, and methods of screening formutations associated with Canavan disease. Guangping Gao, M.D., Ph.D. Page 11 Past Grant Support NAME Guangping Gao DEPARTMENT Pathology and Laboratory Medicine Grant Name & Sponsor Number Period of Award Grant Category* Role in Grant ** Juvenile Diabetes Foundation International 070199 063005 PG AAV Collaboration with ARIAD 041502011403 Gene Therapy for Malignant Mesothelioma Improved Adenoassociated Virus Purification P30DK19525 Diabetes Endocrinology Research Center Creation of Vector Systems for Gene Therapy P30DK47757 Gene Therapy for Cystic Fibrosis and other Genetic Disease DATE 2/15/2016 % Effort Funding Source Annual Direct Cost Additional Comments Project Leader 15% JDRF $164, 671 ($1,388,892) PI: Wilson IG Project Leader 25% ARIAD $293,782 PI: Wilson 051800033105 PP Project Leader 5% NIH/NCI P01 ($1,252,292) PI: Albelda 031502022804 IG PI 5% Puresyn Inc $190,162 040100033103 PP Project Leader 5% NIH/NIDDK $44,000 070100063005 IG Project Leader 10% GSK $235,980 (2,100,000) PI: Wilson 19982003 PP Project Leader NIH/NIDDK $68, 369 PI:Wilson **For Grant Category, use code in bold from the following menu : R01 NIH R01 PP NIH Program Project, Center or Core Grant FG Federal Grants – Other (including other individual NIH grants And grants from VA, NSF, Dept of Energy, etc) grants) CT TG IG PG O PI: Lazar/ Matchinsky Clinical Trials Training Grants Industrial Grants (including pharmaceuticals) Private Foundation Grants (including internal PENN Other * For program projects, specify whether PI, co-leader, or project leader. For center, core and training grants, similarly specify your role.** Include any additional, brief information. For clinical trials, for example, specify if multi-center or single center and indicate role of PENN site. Explain any grants in “Other” category. Guangping Gao, M.D., Ph.D. Page 12 Current Grant Support NAME Guangping Gao DEPARTMENT Pathology and Laboratory Medicine DATE 2/15/2016 Grant Name & Sponsor Number Period of Award Grant Category * Role in Grant ** % Effort Funding Source Annual Direct Cost P01 A15227 HIV-1 Vaccine based on chimp serotypes of adenovirus 040102033107 PP Project Leader 5% NIH/NIAID $205,693 P30 DK47757 Molecular Therapy for CF and Genetic Disease 090103083108 PP Project Leader .6 cal NIH/NIDDK $105,000 ($666,588) PI:Wilson P01 HL051746 Gene Therapy For Cystic Fibrosis 070104063009 PP Project Leader 2.4 cal NIH/NHLBI $188,313 PI:Crystal Po1 HL59407 DNA Viruses as Vectors for Cardiovascular Diseases 070104063009 PP Co-Leader 2.4 NIH/NHLBI $263,034 $1,226,121 PI:Wilson CFF R881 Research Development Program – Component III 120103113006 PG Project Leader 1.2 cal CF Foundation $80,000 ($400,000) PI:Wilson Additional Comments **For Grant Category, use code in bold from the following menu : R01 NIH R01 PP NIH Program Project, Center or Core Grant FG Federal Grants – Other (including other individual NIH grants And grants from VA, NSF, Dept of Energy, etc) grants) CT TG IG PG O Clinical Trials Training Grants Industrial Grants (including pharmaceuticals) Private Foundation Grants (including internal PENN Other * For program projects, specify whether PI, co-leader, or project leader. For center, core and training grants, similarly specify your role. ** Include any additional, brief information. For clinical trials, for example, specify if multi-center or single center and indicate role of PENN site. Explain any grants in “Other” category. Guangping Gao, M.D., Ph.D. Page 13 Pending Grant Support NAME Guangping Gao Grant Name & Sponsor Number Period of Award DEPARTMENT Grant Category* Pathology and Laboratory Medicine Role in Grant ** % Effort Funding Source DATE Annual Direct Cost 2/15/2016 Additional Comments n/a **For Grant Category, use code in bold from the following menu : R01 NIH R01 PP NIH Program Project, Center or Core Grant FG Federal Grants – Other (including other individual NIH grants And grants from VA, NSF, Dept of Energy, etc) grants) CT TG IG PG O Clinical Trials Training Grants Industrial Grants (including pharmaceuticals) Private Foundation Grants (including internal PENN Other * For program projects, specify whether PI, co-leader, or project leader. For center, core and training grants, similarly specify your role. ** Include any additional, brief information. For clinical trials, for example, specify if multi-center or single center and indicate role of PENN site. Explain any grants in “Other” category.