Kenneth G. Cornetta

Kenneth G. Cornetta, MD

Professor of Clinical Medical & Molecular Genetics


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 is part of 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/NHLBI National Gene Vector Biorepository ( which assists investigators in meeting FDA requirements associated with preclinical and clinical gene therapy. Our research looks at way to improve vector production and certification testing.

Key Publications

Shaw, A., and Cornetta, K. Design and Potential of Non-Integrating Lentiviral Vectors. Biomedicines 2014, 2, 14-35.

Gao, H., Hawkins, T., Jasti A., Chen, Y-H., Mockaitis, K., Dinauer, M., Cornetta, K. Development and Evaluation of Quality Metrics for Bioinformatics Analysis of Viral Insertion Site Data Generated Using High Throughput Sequencing. Biomedicines 2014, 2, 195-210.

Carbonaro, D.A., Zhang, L., Jin, X., Montiel-Equihua, C., Geiger, S., Carmo, M., Cooper, A., Fairbanks, L., Kaufman, M. L., Sebire, N. J., Hollis, R. P., Blundell, M. P., Senadheera, S., Fu, P. Y., Sahaghian, A., Chan, R. Y., Wang, X., Cornetta, K., Thrasher, A. J., Kohn, D. B., Gaspar, H. B. Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Molecular Therapy 22:607-22, 2014. PMC3944341

Chodon, T, Comin-Anduix, B., Chmielowski, B., Koya, R.C., Wu, Z., Auerbach, M., Ng, C., Avramis, E., Seja, E., Villanueva, A., McCannel, T.A., Ishiyama, A., Czernin, J., Radu, C.G., Wang, X., Gjertson, D.W., Cochran, A.J., Cornetta, K., Wong, D.J.L., Kaplan-Lefko, P., Hamid, O., Samlowski, W., Cohen, P.A., Daniels, G.A., Mukherji, B., Yang, L., Zack, J.A., Kohn, D.B., Heath, J.R., Glaspy, J.A., Witte, O.N., Baltimore, D., Economou, J.S., and Ribas, A. Adoptive transfer of MART-1 T-cell receptor transgenic lymphocytes and dendritic cell vaccination in patients with metastatic melanoma. Clinical Cancer Research 20:2457-2465, 2014. PMC4070853

Wolstein O., Boyd M., Millington M., Impey H., Boyer J., Howe A., Delebecque F., Cornetta K., Rothe M., Baum C., Nicolson T., Koldej R., Zhang J., Keech N., Camba Colón J., Breton L., Bartlett J., An DS, Chen IS, Burke B., Symonds GP. Preclinical safety and efficacy of an anti-HIV-1 lentiviral vector containing a short hairpin RNA to CCR5 and the C46 fusion inhibitor. Mol Ther Methods Clin Dev. 2014 Feb 12;1:11. doi: 10.1038/mtm.2013.11 PMC4365823

Eissenberg, L.G., Rettig, M. P., Ritchey, J. K., Prior, J., Schwarz, S., Frye, J. White, B. S., Fulton, R., Ghobadi, A., Couriel, D., Seegulam, M. E., Piwnica-Worms, D., Dehdashti, F., Cornetta, K., DiPersio J. F. Infusion and [18F]FHBG PET/CT Imaging of CD34-TK75 Transduced Donor T cells in Patients who Relapse after Allogeneic Stem Cell Transplant: Safety and Feasibility . Molecular Therapy, 23:1110-1122, 2015. PMC4817759

Cornetta, K., Kipsang, S., Gramelspacher, G., Choi, E., Brown, C., Hill, A., Loehrer, P.J., Busakhala, N., Asirwa, F.C. Integration Of Palliative Care Into Comprehensive Cancer Treatment In Western Kenya. J. Global Oncology September 23, 2015, doi: 10.1200/JGO.2015.000125. PMC5551647

Punwani, D., Kawahara, M., Yu, J., Sanford, U., Roy, S., Patel, K., Carbonaro, D. A., Karlen, A. D., Khan, S., Cornetta, K., Rothe, M., Schambach, A., Kohn, D. B., Malech, H. L., McIvor, R. S., Puck, J. A., Cowan, M. J. Lentivirus Medicated Correction of Artemis-Deficient Severe Combined Immunodeficiency. Human Gene Therapy 28:112-124, 2017. PMC pending

Shaw, A. M., Joseph, G. L., Jasti, A. C., Sastry-Dent, L., Witting, S., Cornetta, K. Differences in vector-genome processing and illegitimate integration of non-integrating lentiviral vectors. Gene Ther. 24:12-20, 2017. PMC5269419

Shaw, K.L., Garabedian, E., Mishra, S., Barman, P., Davila, A., Carbonaro, C., Shupien, S. Silvin, C., Geiger, S., Nowicki, B., Smogorzewska, E. M., Brown, B., Wang, X., de Oliveira, S., Choi, Y., Ikeda, A., Terrazas, D. Fu,P-Y., Yu, A., Campo, B., Cooper, A., Engel, B., Podsakoff, G., Balamurugan, A., Anderson, S., Muul, M., Jagadeesh, G.J., Kapoor, N., Tse, J., Moore, T. B., Purdy, K., Rishi, R., Mohan, K., Skoda-Smith, S., Buchbinder, D., Abraham, R. S., Scharenberg, A., Yang, O. O., Cornetta, K., Gjertson, D., Hershfield, M., Sokolic, R., Candotti, F., and Kohn, D. B. A Phase II Clinical Trial of Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency using a g-Retroviral Vector and Reduced Intensity Conditioning. Journal of Clinical Investigation 127:1689-1699, 2017. PMC5409097

Cornetta, K., Duffy, L., Turtle, C.J., Jensen, M., Forman, S., Binder-Scholl, G. Fry, T., Chew, A., Maloney, D. G., and June, C.H. Absence of Replication Competent Lentivirus in the Clinic: Analysis of Infused T Cell Products. Molecular Therapy (in press) doi: 10.1016/j.ymthe.2017.09.008


(317) 274-2240 

Medical & Molecular Genetics
Walther Hall 980 W Walnut Street, R3 C649
Indianapolis, IN 46202-5121


Titles & Appointments

  • Adjunct Clinical Professor of Microbiology & Immunology
  • Adjunct Clinical Professor of Medicine
  • Professor, Adjunct Professor, Adjunct Professor, Graduate Faculty Member w/Endorsement