Cornetta Lab

The research laboratory led by Kenneth G. Cornetta, MD, serves as a national resource for gene therapy work. The research focus of this lab is on using viral-based gene transfer as a means of introducing genetic sequences that will correct mutations and ameliorate disease.

The Cornetta lab is part of the Indiana University Vector Production Facility, established in 1995 to generate clinical gene therapy vector products. To date the lab has certified more than 30 products for Phase I/II trials and the lab team has published extensively on release testing to meet FDA requirements.

Active Research

The Cornetta Lab is the NIH/NHLBI National Gene Vector Biorepository, which assists investigators in meeting FDA requirements associated with preclinical and clinical gene therapy. The research of this lab looks at ways to improve vector production and certification testing.

PI: Kenneth Cornetta
Agency: Indiana CTSI and IU Center for Global Health’s Research Pilot Grant
Period: 5/1/2017 – 4/30/2018
This pilot grant looks at innovating ways to utilize telecommunication to support patients in remote areas wishing to stay at home during end-of-life.

PI: Kenneth Cornetta
Agency: NIH/NHLBI
Type: 9P40HL116242 Period: 6/15/08–3/31/17, supplement providing funding through 9/30/18
This grant provides a repository archive resource for gene therapy investigators.

PI: William S. Goebel
Agency: NIH/NHLBI
Type: R42-HL099150-02A1  07/01/10-06/30/18
The ultimate goal of this work is to commercialize an approved gene therapy based treatment for FANCA.

Recent Publications

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

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

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

Research Team

Kenneth G. Cornetta, MD

Kenneth G. Cornetta, MD

Professor of Clinical Medical & Molecular Genetics