Recent selected publications:

Urbinati F, Hargrove PW, Geiger S, Romero Z, Wherley J, Kaufman ML, Hollis RP, Chambers CB, Persons DA, Kohn DB, Wilber A. Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells. Exp Hematol 2015

Romero Z, Campo-Fernandez B, Wherley J, Kaufman ML, Urbinati F, Cooper AR, Hoban MD, Baldwin K, Lumaquin D, Wang X, Senadheera S, Hollis RP, Kohn DB. The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells. Molecular Therapy — Methods and Clinical Development 2015

Hoban MD, Cost GJ, Mendel MC, Romero Z, Kaufman ML, Joglekar AV, Ho M, Lumaquin D, Gray D, Lill GR, Cooper AR, Urbinati F, Senadheera S, Zhu A, Liu PQ, Paschon DE, Zhang L, Rebar EJ, Wilber A, Wang X, Gregory PD, Holmes MC, Reik A, Hollis RP, Kohn DB. Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells. Blood, 2015

Baldwin K, Urbinati F, Romero Z, Campo-Fernandez B, Kaufman ML,Cooper AR, Masiuk K, Hollis RP, Kohn DB. Enrichment of Human Hematopoietic Stem/Progenitor Cells Facilitates Transduction for Stem Cell Gene Therapy. Stem Cells, 2015

Gschweng EH, McCracken MN, Kaufman ML, Ho M, Hollis RP, Wang X, Saini N, Koya RC, Chodon T, Ribas A, Witte ON, Kohn DB. HSV-sr39TK Positron Emission Tomography and Suicide Gene Elimination of Human Hematopoietic Stem Cells and Their Progeny in Humanized Mice. Cancer Research, 2014

Carbonaro DA, Tarantal AF, Lee CC, Martinez M, Jin X, Wang X, Hardee CL, Geiger S, Kahl CA,Kohn DB. Effects of Vector Backbone and Pseudotype on Lentiviral Vector-mediated Gene Transfer: Studies in Infant ADA-Deficient Mice and Rhesus Monkeys. Molecular Therapy, 2014

Joglekar AV, Stein L, Ho M, Hoban M, Hollis RP, and Kohn DB. Dissecting the mechanism of histone deacetylase inhibitors to enhance the activity of zinc finger nucleases delivered by integrase-defective lentiviral vectors. Human Gene Therapy, 2014

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. Molecular Therapy, 2014.

Oliveira SN, Ryan C, Giannoni F, Hardee C, Tremcinska I, Katebian B, Wherley J, Sahaghian A, Grogan T, Elashoff D, Cooper L, Hollis R, and Kohn DB. Modification of Hematopoietic Stem/Progenitor Cells with CD19-specific Chimeric Antigen Receptors as a Novel Approach for Cancer Immunotherapy. Human Gene Therapy, 2013

Joglekar A, Hollis RP, Kuftinec G, Shenadeera S, Awe JP, Chan R, Reik A, Flinders C, Byrne JA, Holmes MC, Gregory PD, and Kohn DB Integrase-Defective Lentiviral Vectors for Delivery of Zinc Finger Nucleases and Donor Templates for Site-Specific Gene Modification. Molecular Therapy, 2013

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 DBGene transfer to human bone marrow for sickle cell disease. Journal of Clinical Investigation, 2013

Giannoni F, Hardee CL, Wherley J, Gschweng E, Senadheera S, Kaufman ML, Chan R, Bahner I, Gersuk V, Wang X, Gjertson D, Baltimore D, Witte ON, Economou JS, Ribas A, and Kohn DB. Allelic Exclusion and Peripheral Reconstitution by TCR Transgenic T Cells Arising from Transduced Human Hematopoietic Stem/Progenitor Cells.  Molecular Therapy, 2013

De Oliveira SN, Wang J, Ryan C, Morrison SL, Kohn DB, Hollis RP. A CD19/Fc fusion protein for detection of anti-CD19 chimeric antigen receptors. Journal of Translational Medicine, 2013

Candotti F, Shaw K, Muul L, Carbonaro D, Sokolic R, Choi C, Shurman S, Garabedian E, Kesserwan C, Jagadessh GJ, Fu PY, Gschweng E, Cooper A, Tisdale JF, Weinberg KI, Crooks GM, Kapoor N, Shah A, Abdel-Azim H, Yu XJ, Smogorzewska M, Wayne AS, Rosenblatt HM, Davis CM, Hanson C, Rishi RG, Wang X, Gjertson D, Yang OO, Balamurugan A, Bauer G, Ireland J, Engel BC, Podsakoff GM, Hershfield M, Blaese RM, Parkman R, and Kohn DBGene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency: Clinical Comparison of Retroviral Vectors and Treatment Plans. Blood, 2012

Carbonaro DA, Jin X, Wang X, Yu XJ, Rozengurt N, Kaufman ML, Wang X, Gjertson D, Zhou Y, Blackburn MR, Kohn DB. Gene therapy ex vivo in ADA-deficient mice: the role of enzyme replacement therapy and cytoreduction. Blood, 2012

Tarantal AF, Giannoni F, Sumiyoshi T, Lee CI, Hollis RH, Wherley J, Martinez M, Leapley A, Kahl C, Louie SG, and Kohn  DB.  Non-Myeloablative Conditioning Regimen to Increase Engraftment of Gene-Modified Hematopoietic Stem Cells. Molecular Therapy, 2012

Cooper AR, Patel S, Senadheera S, Plath K, Kohn DB, Hollis RP. Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration. Journal of Virological Methods, 2011