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NGVB Facilitated Publications


2008

Daud, A.I., DeConti, R. C., Andrews, S., Urbas, P., Ricker, I., Sondak, V. K., Munster, P.N., Sullivan, D. M., Ugen, K.E., Messina, J. L., Heller, R.. Phase I Trial of Interleukin-12 Plasmid Electroporation in Patients with Metastatic Melanoma. Journal of Clin Oncol., 26:5896-903, 2008 PMC2645111.

Glorioso J.C., Fink. D. J. Herpes Vector –mediated Gene Transfer in the treatment of Chronic Pain. Molecular Ther. 17:13-8. 2008 PMC2613169

Hauswirth, W.W., Aleman, T. S., Kaushal, S., Cideciyan, A. V., Schwartz, S. S. Wang, L., Conlon, T. J., Boye, S. L., Flotte, T. R., Byrne, B. J., Jacobson, S. G.. Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial. Hum Gene Therapy. 19:979-90. 2008. PMID 18774912. Free PMC article PMC2940541

Peters, B., Discherl. S., Dantzer, J., Nowacki, N., Cross S., Cornetta, K., Dinauer, M.C., and Mooney SD. Automated analysis of viral integration sites in gene therapy research using the SeqMap web resource. Gene Ther.15:1294-8, 2008. PMC2766545

Sheets, R. L.,Judith Stein, J., Bailer, R. T., Koup, R. A., Nason, M., He, B., Koo, E., Trotter, H., Duffy, C., Manetz, T. S., Gomez, G. Biodistribution and Toxicological Safety of Adenovirus Type 5 and Type 35 Vectored Vaccines Against Human Immunodeficiency Virus-1 (HIV-1), Ebola, or Marburg Are Similar Despite Differing Adenovirus Serotype Vector, Manufacturer’s Construct, or Gene Inserts. Journal of Immunotoxicol. 5:315-35.2008 PMC2777703

2009

Brantly, M.L., Chulay, J. D., Wang, W., Mueller, C., Humphries, M., Spencer, L. T., Rouhania, F., Conlon, T. J., Calcedo, R., Betts, B., Spencer, S., Byrne, B. J., Wilson, J. M., Flotte, T. R. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. PNAS USA. 106:16363–8, 2009. PMC2752529.

Cideciyan, A. V., Hauswirth, W. W., Aleman, T. S., Kaushal, S., Schwartz, S. B., Boye, S. L., Windsor, E. A. M., Conlon, T. J., Sumaroka, A., Pang, J-J., Roman, A. J., Byrne, B. J., Jacobson, S. G. Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year. Human Gene Thera. 20:999-1004, 2009. PMC2829287

Cornetta, K.,Matheson, L. The National Gene Vector Biorepository Pharm/Tox Database. Mol. Ther. 17(4):582-4. 2009. PMC2835100.

David J. Margolis, D. J., Lee M. Morris, L. M., Maryte Papadopoulos, M., Linda Weinberg, L., Jennifer C. Filip, J. C., Stephanie A. Lang, S. A., Vaikunth S. S., Crombleholme, T. M. Phase I Study of H5.020CMV.PDGF-B to Treat Venous Leg Ulcer Disease. Mol. Ther. 17:1822-9, 2009. PMC2835007

Hammond, H. K. Tang, T. Gene therapy for myocardial infarction–associated congestive heart failure: how far have we got? Dialogues in Cardiovascular Medicine 14:27-51. 2009. PMC3119547.

Hao, S., Wolfe, D., Glorioso, J.C., Mata, M., Fink, D. J.. Effects of transgene-mediated endomorphin-2 in inflammatory pain. European Journal of Pain. 13: 380-6. 2009. PMC2656597

Hollyman DR,Stefanski J, Przybylowski M, Bartido S, Borquez-Ojeda 0, Taylor C, Yeh R, Capacio V, Olszewska M, Hosey J,Sadelain M, Brentjens RJ, Rivière I. Manufacturing validation of biologically functional T cells targeted to CD19 antigen for autologous adoptive cell therapy. J Immunotherapy 32:169–180. 2009. PMC2683970

Mendell, J. R., Rodino-Klapac, L. R., Rosales-Quintero, X., Kota, J., Coley, B. D., Galloway, G., Craenen, J. M., Lewis, S., Malik, V., Shilling, C., Byrne,B. J., Conlon, T., Campbell, K. J., Bremer, W. G., Viollet, L., Walker, C. M. , Sahenk, Z., and Clark, K. R. . Limb-Girdle Muscular Dystrophy Type 2D Gene Therapy Restores alpha-Sarcoglycan and Associated Proteins. Ann Neurol. 66:290-7. 2009 PMID 19798725

Wolfe, D.,Mata M., Fink, D. J.. A Human trial of HSV-mediated gene transfer for the treatment of chronic pain. Gene Ther. 16: 455-60, 2009. PMC2683467.


2010

Flotte, T. R., Fischer, A. C., Goetzmann, J., Mueller, C., Cebotaru, L., Yan, Z., Wang, W., Wilson, J. W., Guggino, W. B., Engelhardt, J. F. Dual Reporter Comparative Indexing of rAAV Pseudotyped Vectors in Chimpanzee Airway. Mol.Ther.18:594-600. 2010. PMC2839428.

Gerrits, A., Dykstra, B., Kalmykowa, O. J., Klauke, K., Verovskaya, E., Broekhuis, M. J. C., de Haan, G., Bystrykh, L. V. Cellular barcoding tool for clonal analysis in the hematopoietic system. Blood 115:2610-8, 2010. PMID20093403

Kochenderfer, J.N., Wilson, W.H., Janik, J.E., Dudley, M.E. Stetler-Stevenson, M.,Feldman, S.A., Maric, I., Raffeld, M., Nathan, D. N., Lanier, B. J., Morgan, R..A., Rosenberg, S. A.  Eradication of B-lineage cells and regression of  lymphoma in a patient treated with autologous T cells genetically-engineered  to recognize CD19.  Blood. 116:4099-4102, 2010. PMC2993617

Siprashvili Z,Nguyen NTA, Bezchinsky MY, Marinkovich VP, Lane At, Khavari PA. Long-Term Type VII Collagen Restoration to Human Epidermolysis Bullosa Skin Tissue. Hum Gene Ther. Oct; 21(10):1299-310. 2010. PMC 2957245

2011

Brentjens RJ, Rivière I, Park JH, Davila ML, Wang X, Stefanski J, Taylor C, Yeh R, Bartido S, Borquez-Ojeda O, Olszewska M, Bernal Y, Pegram H, Przybylowski M, Hollyman D, Usachenko Y, Pirraglia D, Hosey J, Santos E, Halton E, Maslak P, Scheinberg D, Jurcic J, Heaney M, Heller G, Frattini M, Sadelain M. Safety and persistence of adoptively transferred autologous CD19-targeted T cells in patients with relapsed or chemotherapy refractory B-cell leukemias. Blood 118:4817-28.2011. PMC 3208293

Brown, A., Islam, T., Adams, R., Nerle, S.,Kamara, M., Eger, C. Marder, K. Cohen, B., Schifitto, G., & Justin C. McArthur, J. C., Sacktor, N., Pardo, C. A. Osteopontin enhances HIV replication and is increased in the brain and cerebrospinal fluid of HIV-infected individuals. Journal of Neurovirology 17:382-392, 2011. PMC3331788

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. Mol Ther.19:557-66, 2011. PMC3048188.

Di Stasi, A., Tey, S. K, Dotti, G., Fujita, Y., Kennedy-Nasser, A., Martinez, C., Straathof, K., Liu, E., Durett, A. G., Grilley, B., Liu, H., Cruz, C. R., Savoldo, B., Gee, A. P., Schindler, J., Krance, R. A., Heslop, H. E., Spencer, D. M., Rooney, C. M., Brenner, M. K.. Inducible apoptosis as a safety switch for adoptive cell therapy. N Engl J Med. 365:1673-83, 2011. PMC3236370.

Hawkins, T. B., Danzer, J., Peters, B., Dinauer, M. C., Mockaitis, K., Mooney, S., Cornetta, K. Identifying viral integration sites using SeqMap 2.0. Bioinformatics. 27:720-2, 2011. PMC3042184

Gorell, E., Tichey A., and Lane, A., Successful investigational new drug preparation without reinventing the wheel. J Invest Dermatol. 131 (5): 966-8 2011. doi: 10.1038/jid.2011.38.

Louis, C. U., Savoldo, B., Dotti, G., Pule, M., Yvon, E., Myers, G. D., Rossig, C., Russell, H. V. Diouf, O., Liu, E., Liu, H., Wu, M. F., Gee, A. P., Mei, Z., Rooney, C. M., Heslop, H. E., Brenner, M. K. Antitumor activity and long-term fate of chimeric antigen receptor-positive T cells in patients with neuroblastoma. Blood. 118:6050-6, 2011. PMC3234664.

Ma, C., Fan, R., Ahmad, H., Shi, Q., Comin-Anduix, B., Chodon, T., Koya, R. C., Liu, C-C., Kwong, G. A., Radu, C. G., Ribas, A., Heath, J. R. A clinical microchip for evaluation of single immune cells reveals high functional heterogeneity in phenotypically similar T cells. Nature Medicine 17:738-43, 2011. PMC3681612

Savoldo, B., Ramos, C. A., Liu, E., Mims, M. P., Keating, M. J., Carrum, G., Kamble, R. T., Bollard, C. M., Gee, A. P., Mei, Z., Liu, H., Grilley, B., Rooney, C. M., Heslop, H. E., Brenner, M. K., Dotti, G. CD28 costimulation improves expansion and persistence of chimeric antigen receptor-modified T cells in lymphoma patients. J Clin Invest. 121:1822-6, 2011. PMC3083795.


2012

Adair, J. E., Beard, B. C., Trobridge, G. T., Neff, T.,Rockhill, J. K., Silbergeld , D. L., Mrugala, M. M., Hans-Peter Kiem, H-P. Extended Survival of Glioblastoma Patients After Chemoprotective HSC Gene Therapy. Science Transl. 4:133ra57, 2012. PMC3650895

Al-Mehdi, A. B., Pastukh, V. M., Swiger, B. M., Reed, D. J., Patel, M. R., Bardwell, G. C., Pastukh, V. V., Alexeyev, M. F., Gillespie, M. N. Perinuclear mitochondrial clustering creates an oxidant-rich nuclear domain required for hypoxia-induced transcription.Sci Signal. 5:ra47. 2012 PMC3565837

Candotti, F., Shaw, K. L., Muul, L., Carbonaro, D., Sokolic, R., Choi, C., Schurman, S. H., Garabedian, E., Kesserwan, C., Jagadeesh, G. J., Fu, P.Y., Gschweng, E., Cooper, A., Tisdale, J. F., Weinberg, K. I., Crooks, G. M., Kapoor, N., Shah, A., Abdel-Azim, H., Yu, X. J., Smogorzewska, M., Wayne, A. S., Rosenblatt, H. M., Davis, C. M., Hanson, C., Rishi, R. G., Wang, X., Gjertson, D., Yang, O. O., Balamurugan, A., Bauer, G., Ireland, J. A., Engel, B. C., Podsakoff, G. M,. Hershfield MS, Blaese RM, Parkman R, Kohn DB. Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. Blood. 120:3635-46. 2012 PMC3488882

Cioffi, D. L., Wu, S., Chen, H., Alexeyev, M., St Croix, C. M., Pitt, B. R., Uhlig, S., Stevens, T. Orai1 determines calcium selectivity of an endogenous TRPC heterotetramer channel. Circ Res. 110:1435-44. 2012. PMC3388001

Gerrits, A., Walasek, M. A., Olthof, S., Weersing, E., Ritsema, M., Zwart, E., van Os, R., Bystrykh, L. V., de Haan, G. Genetic screen identifies microRNA cluster 99b/let-7e/125a as a regulator of primitive hematopoietic cells. Blood 119:377-87 2012.

Green, M.R., Lockey, T., Mehta, P. K., Kim, Y. S., Eldridge, P. A. W., Gray, J. T., Sorrentino, B. P. Transduction of human CD34+ Repopulating Cells with a Self-inactivating Lentiviral Vector for SCID-X1 Produced at Clinical Scale by a Stable Cell Line. Human Gene Therapy Methods. 23:297-308, 2012. PMC3732136

Heck JN, Ponik SM, Garcia-Mendoza MG, Pehlke CA, Inman DR, Eliceiri KW, Keely PJ. Microtubules regulate GEF-H1 in response to extracellular matrix stiffness. Mol Biol Cell. Jul;23 (13):2583-92 2012. PMC3386221

Leath, A., and Cornetta, K. Developing novel lentiviral vectors into clinical products. Methods in Enzymology, 507:89-108, 2012. PMID: 22365770

Kochenderfer, J.N., Dudley, M.E. , Feldman, S.A. , Wilson, W.H., Spaner, D.E., Maric,  I. Stetler-Stevenson, M., Phan, G.Q. , Hughes, M.S., Sherry, R.M., Yang, J.C. , Kammula, U.S., Devillier, L., Carpenter, R., Nathan, D. N., Morgan, R.A., Laurencot, C., Rosenberg, S.A..  B-cell Depletion and Remissions of Malignancy Along With Cytokine- associated Toxicity in a Clinical Trial of T Cells Genetically-modified to Express an Anti-CD19 Chimeric Antigen Receptor.  Plenary Paper.  Blood. 119: 2709-2720, 2012. PMC3327450

Ochoa, C. D., Alexeyev, M., Pastukh, V., Balczon, R., Stevens, T. Pseudomonas aeruginosa exotoxin Y is a promiscuous cyclase that increases endothelial tau phosphorylation and permeability. J Biol Chem. 287:25407-18. 2012. PMC3408204

Snook., A. E., Magee, M., Marszalowicz, G. P., Schulz, S., Waldman, S. A. "Epitope-targeted cytotoxic T cells mediate lineage-specific antitumor efficacy induced by the cancer mucosa antigen GUCY2C." Cancer Immunol Immunother. 61:713-23. 2012. PMID: 22057677


2013

Aachoui, Y., Leaf, I. A., Hagar, J. A., Fontana, M. F., Dampos, C. G., Zak, D. E., Tan, M. H., Cotter, P. A. , Vance, R. E. , Aderem, A., Miao, E. A. Caspase-11 Protects Against Bacteria That Escape the Vacuole. Science. 339:975-8. 2013 PMC 3697099

Adler JJ, Heller BL, Bringman LR, Ranahan WP, Cocklin RR, Goebl MG, Oh M, Lim HS, Ingham RJ, Wells CD. Amot130 adapts atrophin-1 interacting protein 4 to inhibit yes-associated protein signaling and cell growth. J Biol Chem. May 24;288(21):15181-93.2013. PMC3663538

 

Adler JJ, Johnson DE, Heller BL, Bringman LR, Ranahan WP, Conwell MD, Sun Y, Hudmon A, Wells CD. Serum deprivation inhibits the transcriptional co-activator YAP and cell growth via phosphorylation of the 130-kDa isoform of Angiomotin by the LATS1/2 protein kinases. Proc Natl Acad Sci U S A. Oct 22 110:(43):17368-73.2013. PMC3808603

 

Aloia, A., Duffy, L., Pak, V., Lee, K., Sanchez-Martinez, S., Derse, D., Heidecker, G., Cornetta, K., and Rein, A. A reporter system for replication-competent gammaretroviruses: the inGluc-MLV-DERSE assay. Gene Therapy 20:169-176, 2013. PMC 3374051

 

Alzoubi A, Almalouf P,Toba M, O’Neill K, Qian X, et al. TRPC4 Inactivation confers a survival benefit in severe pulmonary arterial hypertension. Am J Pathol 183 (6):1779-1788 2013. PMC5745549

 

Babu R, and Brown A. A consensus surface activation mrker signature is partially dependent on human immunodeficiency virus type 1 Nef expression within productively infected macrophages. Retrovirology Dec16; 10:155. 2013. PMC3883119

Balczon R, Prasain N, Ochoa C, Praer J, Zhu B, et al. Pseudomonas aeruginosa exotoxin Y-mediated tau hyperphosphorylatin impairs microtubule assembly in pulmonary microvascular endothelial cells. PLoS One Sept 4; 8(9):e74343, 2013. PMC3762819

Boeke, A., Dumas, P., Reeves, L., McClurg, K., Bischof, D., Sego, L., Auberry, A., Tatikonda, M., Cornetta, K. Vector Production in An Academic Environment: A Tool to Assess Production Costs. Human Gene Therapy Methods. 24: 49-57, 2013. PMC4015083

 

Brentjens R.J,Davila M.L, Riviere I, Park J, Wang X, L. G. Cowell L.G, Bartido S, Stefanski JTaylor C, Olszewska M, Borquez-Ojeda O, Qu J,Wasielewska T, He Q, Bernal Y, Rijo I.V. Hedvat C,Kobos R, Curran K, Steinherz P, Jurcic J,Rosenblat T, Maslak P, Frattini M, Sadelain M. CD19-Targeted T Cells Rapidly Induce Molecular Remissions in Adults with Chemotherapy-Refractory Acute Lymphoblastic Leukemia. Sci. Transl. Med. 5, 177ra38. 2013.PMC3742551

 

Cornetta, K., Tessanne, K., Long, C., Yao, J., Satterfield, C., Westhusin, M. Transgenic sheep generated by lentiviral vectors: Safety and integration analysis of surrogates and their offspring. Transgenic Research 22:737-45, 2013. PMC3606283

 

Grupp, S A, Kalos, M., Barrett, D., Aplenc, A., Porter, D. L., Rheingold, S. R.., Teachey,D. T. ., Chew, A., Hauck, B., Wright, J. F.., Milone, M.C., Levine, B. L., June, C. Chimeric Antigen Receptor-Modified T Cells for Acute Lymphoid Leukemia. N.ENGL J Med. 18;368(16):1509-18, 2013. PMC4058440

Katwal, A. B.,, Konkalmatt, P. R., Piras, B.A., Hazarika, S., Li, S. S., Lye, R. J., Sanders, J. M., Ferrante, E. A., Yan, Z., Annex, B. H., French, B. A. Adeno-associated virus serotype 9 efficiently targets ischemic skeletal muscle following systemic delivery. Gene Therapy 20:930-8, 2013. PMC 3758463.

Kochenderfer, J.N., Dudley,M.E., Carpenter, R.O., Kassim, S.H., Rose, J.J., Telford, W.G., Hakim, F.T., Halverson, D.C., Fowler, D.H., Hardy, N.M., Mato, A.R., Hickstein, D.D., Gea-Banacloche, J.C., Pavletic, S.Z., Sportes, C., Maric, I., Feldman, S.A., Hansen, B.G., Wilder, J.S., Blacklock-Schuver, B., Jena, B., Bishop, M.R., Gress* R.E.,* Rosenberg*, Steven A.,.(co-senior authors). Donor-derived CD19-targeted T cells cause regression of malignancy persisting after allogeneic hematopoietic stem cell transplantation. Blood: 2013 122: 4129-4139. PMC 3862276

Lin, J.Y. Production and validation of recombinant adeno-associated virus for channel rhodopsin expression in neurons. Methods in Molecular Biology Ion channels: methods and protocols. 998:401-15, 2013.

Lin J.Y., Knutsen P.M., Muller A., Kleinfeld D. and Tsien R.Y. ReaChR: A red-shifted variant of channelrhodopsin enables deep transcranial optogenetic excitation. Nature Neuroscience. 16:1499-508, 2013. PMC3793847

Lin J.Y., Sann S.B., Zhou K., Nabavi S., Proulx C., Malinow R., Jin Y. and Tsien R.Y.. Optogenetic inhibition of synaptic release with chromophore-assisted light inactivation (CALI). Neuron. 79:241-53, 2013. PMC3804158

Nguyen, T., Rich, A., Dahl, R. MiR-24 Promotes the Survival of Hematopoietic Cells. PLoS One. 8:e55406. 2013. PMC3559586

O’Reilly, Marina, Kohn, Donald B., Bartlett,Jeffrey, Benson, Janet, Brooks, Philip J., Byrne, Barry J, Camozzi, Carlos, Cornetta, Kenneth, Crystal, Ronald G., Fong, Yuman, Gargiulo, Linda, Gopal-Srivastava, Rashmi, High, Katherine A.,Jacobson, Samuel G., Jambou, Robert C.,

Montgomery, Maureen, Rosenthal, Eugene,Samulski, R. Jude, Skarlatos, Sorrentino, Wilson, James M., Xie, Yun, and Corrigan-Curay, Jacqueline. Gene Therapy for Rare Diseases:

Summary of a National Institutes of Health Workshop, September 13, 2012. Human Gene Therapy 24:355-362 2013. PMC3631014

 

Parra-Bonilla G, Alvarez DF, Alexeyev M, Vasauskas A, Stevens T. Lactate dehydrogenase a expression is necessary to sustain rapid angiogenesis of pulmonary microvascular endothelium. PLoS One Sep 26;8(9):e75984 2013. PMC3784391

Ponik SM, Trier SM, Wozniak MA, Eliceiri KAW, Keely PJ. RhoA is down-regulated at cell-cell contacts via p190RhoGAP-B in response to tensional homeostasis. Mol Biol Cell. Jun; 24(11):1688-99, 2013. PMC3667722

Romero Z, Urbinati F, Geiger S, Cooper A, Wherley J, Kaufman ML, Hollis R, Senadheera S, Ruiz de Assin R, Sahagian A, Wang X, Gjertson D, DeOliveira S, Kempert P, Shupien S, Abdel-Azim H, Walters M, Meiselman HJ, Marder V, Coates TD, and Kohn DB. Beta globin Gene transfer to human bone marrow for sickle cell disease. J Clin Invest, 123:3317–3330,Jul 1 2013. PMCID: PMC4011030

 

Shokolenko IN, Fayzulin RZ, Katyal S, McKinnon PJ, Wilson GL, et al. Mitochondrial DNA ligase is dispensable for the viability of cultured cells but essential for mtDNA maintenance. J. Biol Chem 288 (37):26594-26605 2013. PMC3772206

Shokolenko IN, Wilson GL, Alexeyev MF. Persistent damage induces mitochondrial mitochondrial DNA degradation. DNA Repair (Amst) 12:488-499. 2013. PMC3683391

Struckhoff, A.P., Rana, M.K., Kher, S. S., Burow, M. E., Hagan, J. L., Del Valle, L., Worthylake, R. A. PDZ-RhoGEF (PRG) is essential for CXCR4-driven breast tumor cell motility through spatial regulation of RhoA. J. Cell Sci. 126:4514-26, 2013 PMC3784824

Watson, R. S., Broome, T. A., Rice, B. L., Kay, J. D.,, Smith, A. D., Gouze, E., Gouze, J-N, Levings, P. P., Dacanay, A., Hauswirth, W.W., Nickerson, D. M., Dark, M., Colahan, P. T., Ghivizzani, S.C. scAAV-mediated gene transfer of interleukin-1 receptor antagonist to synovium and articular cartilage in large mammalian joints. Gene Ther. 20:670-7. 2013. PMC3577988

Wood, T. R., Chow, R. Y., Hanes C. M., Zhang, X., Kashiwagi, K., Shirai, Y., Trebak, M., Loegering, D. J., Saito, N., Lennartz, M. R.. PKC-{varepsilon} pseudosubstrate and catalytic activity are necessary for membrane delivery during IgG-mediated phagocytosis. J Leukoc Biol. 94:109-22, 2013. PMC3685018

 

 

2014

 

Boulad F,Wang X, Qu J,Taylor C,Ferro L, Karponi G, Bartido S, Giardina P,Heller G, Prockop S.E,Maggio A,Sadelain M and Rivière I.  Safe mobilization of CD34+ cells in adults with beta-thalassemia and validation of effective globin gene transfer for clinical investigationBlood Mar 6;123(10): 1483-6 2014. PMID: 24429337. PMC3945860.

Chodon T, Begona C,Bartosz C, Koya R, Zhongqi W, Martin A, Ng C,Avramis E, Seja E, Villanueva A, McCannel T, Ishiyama A, Czernin, J, Radu G, Wang X, Gjertson D, Cochran A, Cornetta K, Wong D, Kaplan-Lefko, P, Hamid O, Samlowski W, Cohen P, Daniels G, Mukherji B, Yang L, Zaqck J, Kohn D, Heath J, Glaspy J, Witte O, Baltimore D, Economou J, and Ribas A. Adoptive transfer of MART-1 T cell receptor transgenic lymphocytes and dendritic cell vaccination in patients with metastatic melanoma. Clin Cancer Res. doi:10.1158/1078-0432.CCR-13-3017 published online Mar 2014. PMID: 24634374. PMC 4070853

 

Chodon T, Comin-Anduix B, Chmielowski B, Koya K, Wu Z, Allen-Auerbach M, Ng C, Avramis E, Seja E, Villanueva A, McCannel T, Ishiyama A, Czernin J, Radu C, Wang X, Gjertson D, Cochran A, Cornetta K, Wong D, Kaplan-Lefko P, Hamid O, Samlowski W, Cohen P, Daniels G, Mukherji B, Yang L, Zack J, Kohn D, Heath J, Glaspy J, Witte O, Baltimore D, Economou J and Ribas A.  2014.  Adoptive transfer of MART-1 T cell receptor transgenic lymphocytes and dendritic cell vaccination in patients with metastatic melanoma.  Clin Cancer Res.  20:2457-65. PMCID: PMC4070853

 

 

Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB. Pre-clinical demonstration of lentiviral vector mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Mol Ther.Mar 22(3):607-22.2014. PMC:3944341

 

 

Davila M.L, Riviere I, Wang X , Bartido S,Park J, Curran K,Chung S.S, Stefanski J, Borquez-Ojeda O, Olszewska M, Qu J Wasielewska T,He Q,Fink M,Shinglot H Youssif M,Satter M, Wang Y, Hosey J,Quintanilla H,Halton E, Bernal Y,Bouhassira D.C.G,Arcila M.E, Gonen M, Roboz G.J Maslak P, Douer D,Frattini M.G., Giralt S, Sadelain M, Brentjens R. Efficacy and Toxicity Management of 19-28z CAR T Cell Therapy in B Cell Acute Lymphoblastic Leukemia. Sci. Transl. Med. 6, 224ra25. 2014. Doi:10.1126/scitranslmed.3008226. PMC 4684949

Eissenberg, LG, Rettig, M, Dehdashti, F, Piwnica-Worms, D, and DiPersio,JF, (2014). Suicide genes:monitoring cells in patients with a safety switch. Front Pharmacol 6, 5:241.PMID: 25414668. PMC 4222135

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(2), 195-210; PMC5423470

Hacein-Bey-Abina, S., Pai, S.-Y. Gaspar, H.B., Armant, M. Berry, C.C., Blanche, Bleesing, J.,Blondeau, H. de Boer, K.F. Buckland, L. Caccavelli,. Cros, G.m, De Oliveira, S., Fernández, K.S., Guo, D.,Harris, C.E.,Hopkins, G, Lehmann,L.E., A. Lim, W.B. London, J.C.M. van der Loo, N. Malani, F. Male, Malik, P., Marinovic, M.A, McNicol, A.-M.,Moshous,D. Neven, B. Oleastro, M., Picard,C., Ritz, J.,C. Rivat, Schambach,A., Shaw,K.L., Sherman, E.A.,Silberstein, L.E., Six, E., Touzot, F., Tsytsykova, A., Xu-Bayford, J., Baum, C., Bushman, F.D., Fischer, A., Kohn, D.B., Filipovich, A.H., Notarangelo, L.D., Cavazzana, M., Williams, D.A.,and Thrasher, A.J. A Modified γ-Retrovirus Vector for X-LinkedSevere Combined Immunodeficiency. N. ENGL J MED 2014;371:1407-17..Oct 9 2014. PMC 4274995


Kochenderer, J.N., Dudley, M.E.,Kassim, S.H., Somerville, R.P.T., Carpenter, R.O., Stetler-Stevenson, M., Yang, J.C.,Phan, G.Q., Hughes, M.S., Sherry, R.M., Raffeld, M., Feldman, S., Lu, L., Li, Y.F., Ngo, L.T., Goy, A., Feldman, T., Spaner, D.E., Wang, M.L., Chen, C.C., Kranick, S.M., Nath, A., Nahtan, D.N., Morton, K.E., Toomey, M.A., Rosenberg, S.A., Chemotherapy-refractory Diffuse Large B-cell Lymphoma and Indolent B-cell Malignancies Can be Effectively Treated with Autologous T Cells Expressing an Anti-CD19 Chimeric Antigen Receptor. Rapid Communication Article. Journal of Clinical Oncology. 2014 Aug 25. PMC4322257

 

Shokolenko IN, Wilson GL, Alexeyev MF. The “Fast” and “slow” Modes of Mitochondrial DNA Degradation: Mitochondrial DNA. Epub 2014. Mitochondrial DNA A DNA Mapp Seq Anal. 27:490-8, 2016. PMC4333107

Xu N, Cioffi DL, Alexeyev M, Rich TC, Stevens T. Sodium Entry Through Endothelial Store Operated Calcium Entry Channels: Regulation by Orai1. Am J Physiol Cell Physiol. 308(4): C277–C288, 2015. Epub 2014. PMC 4329424


2015

Ahmed N, Brawley VS, Hegde M, Robertson C, Ghazi A, Gerken C, et al. Human Epidermal Growth Factor Receptor 2 (HER2) -Specific Chimeric Antigen Receptor-Modified T Cells for the Immunotherapy of HER2-Positive Sarcoma. J Clin Oncol. 33(15):1688-96, 2015. PMC4429176

Chang E, Liu H, West JA, Zhou X, Dakhova O, Wheeler DA, et al. Clonal Dynamics in vivo of Virus Integration Sites of T cells expressing a safety switch. Mol Ther. 24(4): 736–745, 2016. Epub 2015. PMC4886929

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; 23:1110-22, 2015 PMC4817759

Fayzulin, R.Z.,Perez, M., Kozhukhar, N., Spadafora,D., Wilson, G.L., and Alexeyev, M.F. A method for mutagenesis of mouse mtDNA and a resource of mouse mtDNA mutations for modeling human pathological conditions. Nucleic Acids Res. 43: e62, 2015. PMC4482060

Jian, M. Y., Liu, Y., Li, Q., Wolkowicz, P., Alexeyev, M., Zmijewski, J.W., and Creighton, J.R. N-cadherin Coordinates AMP Kinase Mediated Lung Vascular Repair. AM.J. Physiol. Lung Cell Mol. Physiol., 310: L71–L85, 2015. Epub 2015. PMC4698433

Kozhukhar, N., Spadafora, D., Fayzulin, R., Shokolenkom, I.N., and Alexeyev, M. The efficiency of the translesion systheseis across abasic sites by mitochondrial DNA polymerase is low in mitochondria of 3T3 cells. Mitochondrial DNA, Oct 16: 1-7, 2016. Epub 2015PMC4834068

Lin Z, Zhou P, von Gise A, Gu F, Ma Q, Chen J, Guo H, van Gorp PR, Wang DZ, Pu WT. Pi3kcb links Hippo-YAP and PI3K-AKT signaling pathways to promote cardiomyocyte proliferation and survival. Circ Res. 116:35-45, 2015. Epub 2014 PMC4282610

Nguyen, J.M., Qualmann, K.J., Okashah, R., Reilly, A., Alexeyev, M.F., and Campbell, D.J. 5p deletion: Current knowledge and future directions. Am. J. Med. Genet. C Semin. Med. Genet. 169, 224-238, 2015. PMC4736720

Prendiville TW, Guo H, Lin Z, Zhou P, Stevens SM, He A, VanDusen N, Chen J, Zhong L, Wang DZ, Gao G, Pu WT. Novel Roles of GATA4/6 in the Postnatal Heart Identified through Temporally Controlled, Cardiomyocyte-Specific Gene Inactivation by Adeno-Associated Virus Delivery of Cre Recombinase. PLoS One. 10:e0128105. 2015. PMC 4449121

Shokolenko, I.N., and Alexeyev, M.F. Mitochondrial DNA: A disposable genome. Biochem Biophys. Acta 1852, 1805-1809, 2015. PMC4523420

Xu, N., Cioffi, D.L., Alexeyev, M., Rich, T.C., and Stevens, T. Sodium entry through endothelial store-operated calcium entry channels: regulation by Orai1. AM.J. Physiol. Cell Physiol. 308, C277-288, 2015. PMC4329424

Zhou X, Dotti G, Krance RA, Martinez CA, Naik S, Kamble RT, et al. Inducible caspase-9 suicide gene controls adverse effects from alloreplete T cells after haploidentical stem cell transplantation. Blood. 125(26):4103-13, 2015. PMC4481597.

Zhou X, Naik S, Dakhova O, Dotti G, Heslop HE, Brenner MK. Serial activation of the inducible caspase 9 safety switch after human stem cell transplantation. Mol Ther. 24: 823–831, 2015. PMC4886936


2016

Brudno, J.N., Somerville, R.P.T., Shi, V., Rose, J.J., Halverson, D.C., Fowler, D.H., Gea-Banacloche, J.C., Pavletic, S.Z., Hickstein, D.D., Lu, T.L., Feldman, S.A., Iwamoto, A.T., Kurlander, R., Maric, I., Goy, A., Hansen, B.G., Wilder, J.S., Blacklock-Schuver, B., Hakim, F.T., Rosenberg, S.A., Gress, R.E. and Kochenderfer, J.N., Allogeneic T cells that express an anti-CD19 chimeric antigen receptor induce remissions of B-cell malignancies that progress after allogeneic hematopoietic stem-cell transplantation without causing graft-versus-host disease Journal of Clinical Oncology 34: 1112-1121, 2016. PMC4872017


Chtarto, A, Humbert-Claude, M. and Bockstael1,O, Das, A. T, Boutry, S., Breger, L. S.,

Klaver, B., Melas, C., Barroso-Chinea, P, Gonzalez-Hernandez, T.,Muller, R.N., DeWitte, O.,

Levivier, M., Lundberg, C., Berkhout, B., and Tenenbaum, L., A regulatable AAV vector mediating GDNF biological effects at clinically-approved sub-antimicrobial doxycycline doses. Mol Ther. (2016), 5,16027, doi 10.1038/mtm.2016.27. PMC4813607

 

Gardner, R., Wu D., Cherian, S., Fang, M., Hanafi, L., Finney, O., Smithers, H., Jensen, M.C., Riddell, S.R., Maloney, D.G., and Turtle, C.J. Acquisition of a CD19 negative myeloid phenotype allows immune escape of MLL-rearranged B-ALL from CD19 CAR-T cell therapy. Blood. 127:2406-10, 2016. PMC4874221

 

Ramos CA, Savoldo B, Torrano V, Ballard B, Zhang H, Dakhova O, Liu E, Carrum G, Kamble RT, Gee AP, Mei Z, Wu MF, Liu H, Grilley B, Rooney CM, Brenner MK, Heslop HE, Dotti G. Clinical responses with T lymphocytes targeting malignancy-associated kappa light chains. J Clin Invest. 126:2588-96, 2016. PMC4922690.

 

Rosario AM, Cruz PE, Ceballos-Diaz C, Strickland MR, Siemienski Z, Pardo M, Schob KL, Li A, Aslanidi GV, Srivastava A, Golde TE, Chakrabarty P. Microglia-specific targeting by novel capsid-modified AAV6 vectors. Mol Ther Methods Clin Dev. 2016 Apr 13;3:16026. doi: 10.1038/mtm.2016.26. eCollection 2016. PMC4909093

Turtle CJHanafi LABerger CGooley TACherian SHudecek MSommermeyer DMelville KPender BBudiarto TMRobinson ESteevens NNChaney CSoma LChen XYeung CWood BLi DCao JHeimfeld SJensen MCRiddell SRMaloney DG. CD19 CAR-T cells of defined CD4+:CD8+ composition in adult B cell ALL patients. J Clin Invest. 2016 Jun 1, 126(6):2123-38. doi: 10.1172/JCI85309. Epub 2016 Apr 25. PMC4887159

 

Turtle, C. J., L.A. Hanafi, C. Berger, T.A. Gooley, S. Cherian, M. Hudecek, D. Sommermeyer, K. Melville, B. Pender, T.M. Budiarto, E. Robinson, N.N. Steevens, C. Chaney, L. Soma, X. Chen, C. Yeung, B. Wood, D. Li, J. Cao, S. Heimfeld, M.C. Jensen, S.R. Riddell and D.G. Maloney. CD19 CAR-T cells of defined CD4+: CD8+ composition for adult B-cell ALL. The Journal of Clinical Investigation. 126:2123-38, 2016. PMCID: PMC4887159

Turtle, C. J. L.A. Hanafi, C. Berger, M. Hudecek, B. Pender, E. Robinson, R. Hawkins, C. Chaney, S. Cherian, X. Chen, L. Soma, B. Wood, D. Li, S. Heimfeld, S.R. Riddell, D.G. Maloney. Immunotherapy of non-Hodgkin lymphoma with a defined ratio of CD8+ and CD4+ CD19-specific chimeric antigen receptor-modified T cells. Sci Trans Med. 8(355), 355ra116, 2016. PMC5045301

Yeku O, Slovin SF. Immune Therapy for Prostate Cancer. Cancer J. 22:334-341, 2016. PMC5117426

2017

Ahmed N,  Brawley V,  Bielamowicz K, Hegde M,  Kalra M, Landi D, Robertson C, Gray TL, Diouf O, Wakefield A, Ghazi A, Gerken C, Yi Z, Ashoori A, Wu M-F, Liu H, Rooney CM, Dotti G, Gee A, Su J,  Kew Y, Baskin D, Zhang YJ, New P, Grilley B, Stojakovic M, Hicks J, Powell SZ, Brenner MK, Heslop HE, Grossman R, Wels WS, Gottschalk S. HER2-specific Chimeric Antigen Receptor–modified virus-specific T-cells for progressive Glioblastoma: a Phase I dose-escalation Trial. JAMA Onc 3:1094-1101 PMC5747970


Ali, S.A., Shi, V., Maric, I., Wang, M., Stroncek, D.F., Rose, J.J., Brudno, J.N., Stetler-Stevenson, M., Feldman, S.A., Hansen, B.G., Fellowes, V.S., Hakim, F.T., Gress, R.E. and Kochenderfer, J.N., T cells expressing an anti-B-cell maturation antigen chimeric antigen receptor cause remissions of multiple myeloma Blood 128: 1688-1700, 2016. PMC5043125

Brudno, J.N., Somerville, R.P.T., Shi, V., Rose, J.J., Halverson, D.C., Fowler, D.H., Gea-Banacloche, J.C., Pavletic, S.Z., Hickstein, D.D., Lu, T.L., Feldman, S.A., Iwamoto, A.T., Kurlander, R., Maric, I., Goy, A., Hansen, B.G., Wilder, J.S., Blacklock-Schuver, B., Hakim, F.T., Rosenberg, S.A., Gress, R.E. and Kochenderfer, J.N., Allogeneic T cells that express an anti-CD19 chimeric antigen receptor induce remissions of B-cell malignancies that progress after allogeneic hematopoietic stem-cell transplantation without causing graft-versus-host disease Journal of Clinical Oncology 34: 1112-1121, 2016. PMC4872017

Cooper AR, Lill GR, Shaw K, Carbonaro-Sarracino DA, Davila A, Sokoic R, Candotti F, Pellegrini M, Kohn DB. Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients. Blood 129:2624-2635, 2017, PMC5428461

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 28:280-288, 2018. Epub 2017 PMC5762981

Heczey A, Louis CU, Savoldo B, Dakhova O, Durett A, Grilley B, Liu H, Wu MF, Mei Z, Gee A, Mehta B, Zhang H, Mahmood N, Tashiro H, Heslop HE, Dotti G, Rooney CM, Brenner MK. CAR T Cells Administered in Combination with Lymphodepletion and PD-1 Inhibition to Patients with Neuroblastoma. Mol Ther. 25:2214-2224, 2017. PMC5589058.

Ramos CA, Savoldo B, Torrano V, Ballard B, Zhang H, Dakhova O, Liu E, Carrum G, Kamble RT, Gee AP, Mei Z, Wu MF, Liu H, Grilley B, Rooney CM, Brenner MK, Heslop HE, Dotti G. Clinical responses with T lymphocytes targeting malignancy-associated kappa light chains. J Clin Invest. 126:2588-96, 2016. PMC4922690.

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 KL, Garabedian E, Mishra S, Barman P, Davila A, Carbonaro D, Shupien S, Silvin C, Geiger S, Nowicki B, Smogorzewska EM, Brown B, Wang X, de Oliveira S, Choi Y, Ikeda A, Terrazas D, Fu PY, Yu A, Fernandez BC, Cooper AR, Engel B, Podsakoff G, Balamurugan A, Anderson S, Muul L, Jagadeesh GJ, Kapoor N, Tse J, Moore TB, Purdy K, Rishi R, Mohan K, Skoda-Smith S, Buchbinder D, Abraham RS, Scharenberg A, Yang OO, Cornetta K, Gjertson D, Hershfield M, Sokolic R, Candotti F, Kohn DB. Clinical Efficacy of gene-modified stem cells in adenosine deaminase-deficieint immunodeficiency. J Clin Invest. 127:1689-1699, 2017 Mar 27.:PMC5409097