Kenneth Cornetta, M.D.

Professor of Clinical Medical and Molecular Genetics
  • B.S. 1978 - State University of New York; Albany NY
  • M.D. 1982 - Albany Medical College; Albany, NY
  • Internal Medicine Residency 1982-85, Indiana University
  • Chief Medical Resident 1985-86, Indiana University
  • NRSA Fellow, NHLBI, NIH 1986-89. Lab of Dr. W, French Anderson
  • Hematology Fellow, 1989-91. University of Wisconsin

The goal of the Cornetta laboratory is to serve as a national resource for gene therapy work. Our research focus is on using viral based gene transfer as a means of introducing genetic sequences that will correct mutations and ameliorate disease. Our lab includes the Indiana University Vector Production Facility that was established in 1995 with the goal of generating clinical gene therapy vector products. To date it has certified over 30 products for Phase I/II trials. We have published extensively on release testing to meet FDA requirements. We are currently the NIH/NCRR National Gene Vector Biorepository for the NCRR ( and the lentiviral production site for the NIH/NHLBI Gene Therapy Resources Program ( Our laboratory is currently developing novel vector pseudotypes for transduction of hematopoietic cells. This work includes vector modification and novel large-scale production and certification methods. In addition, the laboratory is evaluating novel non-integrating vectors for use in cell re-programming. Finally, the laboratory has been evaluating high-throughput sequencing technology for evaluating lentiviral and retroviral integration sites analysis in collaboration with the Center for Genomic and Bioinformatics in Bloomington, IN. This includes development of novel publically available bioinformatics tools to facilitate analysis of integration site studies.


Sadat, M.A.,  Dirscherl,, S., Sastry,, L., Dantzer,, J., Pech,, N., Griffin,, S., Hawkins,, T., Zhao,, Y., Barese,, C. N., Cross,, S., Orazi,, A., An,, C., Goebel,, W. S., Yoder,, M.C., Li,, X., Grez,, M., Cornetta,, K., Mooney,, S.D, Dinauer, M.C. Comparison of gamma-retroviral vector integration in post-transplant hematopoiesis in mice conditioned with either submyeloablative or ablative irradiation. Gene Therapy 16: 1452-64, 2009. PMC2795029

Cavazzana-Calvo , M, Payen , E., Negre , O., Wang , G., Hehir , K., Fusil, F., Down, J., Denaro , M., Brady, T., Westerman, K., Cavallesco , R., Gillet-Legrand, B., Caccavelli, L., Sgarra, R., Maouche-Chrétien, R., Bernaudin, R., Girot, R., Dorazio, R., Mulder, GJ., Polack, A., Bank, A., Soulier, J., Larghero, J., Kabbara, N., Dalle, B., Gourmel, B., Socié, G., Chrétien, S., Cartier, N., Aubourg, P., Fischer, A., Cornetta, K., Galacteros, F., Beuzard, Y., Gluckman, E., Bushman, B., Hacein-Bey Abina, S.   Transfusion independence and HMGA2 activation after gene therapy of human β-thalassemia  Nature 467:318-323, 2010 [PubMed – in process]

Ha, S.P., Klemen, N.P., Brandmaier, A.G., Kinnebrew, G.H., Marsh, J., Hangoc, G., Palmer, D.C., Restifo, N.D., Cornetta, K., Broxmeyer, H.E., and Touloukian, C.E. Tumor destruction and spontaneous autoimmunity following long-term lentiviral CD4+ TCR gene-modified HSC transplantation. Journal of Clinical Investigation 120: 4273-88, 2010. PMC2993591

Feldman, S.A., Goff, S.L., Xu, H., Black, M.A., Kochenderfer, J.N., Johnson, L.A., Yang, J.C., Wang, Q., Parkhust, M.R., Cross, S.B., Morgan, R.A., Cornetta, K., Rosenberg, S.A.  Rapid production of clinical-grade gammaretroviral vectors in expanded surface roller bottles using a “modified” step-filtration process for clearance of packaging cells. Human Gene Therapy 22: 107-115, 2011 [PubMed – in process]

Nelson RP Jr, Yu M, Schwartz JE, Robertson MJ, Hromas R, Fausel CA, Vance GH, Dlouhy SR, Baute JA, Cox EA, Wood LL, Srivastava S, Robertson KA, Haut PR, Farag SS, Abonour R, Cornetta K, Cripe LD. Long-term disease-free survival after nonmyeloablative cyclophosphamide/fludarabine conditioning and related/unrelated allotransplantation for acute myeloid leukemia/myelodysplasia. Bone Marrow Transplant. 45: 1300-1308, 2011. [PubMed – in process]

Cornetta, K., Yao, J., Jasti, A., Koop, S., Douglas, M., Hsu, D., Couture, L.A., Hawkins, T., Duffy, L. Replication Competent Lentivirus Analysis of Clinical Grade Vector Products, Molecular Therapy, 19: 557-566, 2011. PMID: 21179010

Hawkins TB, Dantzer J, Peters B, Dinauer MC, Mockaitis K, Mooney S, and Cornetta, K. Identifying viral integration sites using SeqMap 2.0. Bioinformatics 27; 720-722, 2011. PMID:21245052

Cornetta, K. and Meslin, E.M.  Ethical and scientific issues in gene therapy and stem cell research.  In: Chadwick, ten Have, and Meslin, ed.  Sage handbook series: Health care ethics in an era of globalization.  Sage Publications

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