De Oliveira Satiro N, Wang Jiexin, Ryan Christine, Morrison Sherie L, Kohn Donald B, Hollis Roger P, A CD19/Fc fusion protein for detection of anti-CD19 chimeric antigen receptors. Journal of translational medicine. 2013; 11(2): 23.

Giannoni Francesca, Hardee Cinnamon L, Wherley Jennifer, Gschweng Eric, Senadheera Shantha, Kaufman Michael L, Chan Rebecca, Bahner Ingrid, Gersuk Vivian, Wang Xiaoyan, Gjertson David, Baltimore David, Witte Owen N, Economou James S, Ribas Antoni, Kohn Donald B, Allelic Exclusion and Peripheral Reconstitution by TCR Transgenic T Cells Arising From Transduced Human Hematopoietic Stem/Progenitor Cells. Molecular therapy : the journal of the American Society of Gene Therapy. 2013; 21(5): 1044-54.

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 Sonia I, Sorrentino Brian, Wilson James M, Xie Yun, Corrigan-Curay Jacqueline, Gene therapy for rare diseases: summary of a national institutes of health workshop, september 13, 2012. Human gene therapy. 2013; 24(4): 355-62.

Harrison Frank, Yeagy Brian A, Rocca Celine J, Kohn Donald B, Salomon Daniel R, Cherqui Stephanie, Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis. Molecular therapy : the journal of the American Society of Gene Therapy. 2013; 21(2): 433-44.

McCracken Melissa N, Gschweng Eric H, Nair-Gill Evan, McLaughlin Jami, Cooper Aaron R, Riedinger Mireille, Cheng Donghui, Nosala Christopher, Kohn Donald B, Witte Owen N, Long-term in vivo monitoring of mouse and human hematopoietic stem cell engraftment with a human positron emission tomography reporter gene. Proceedings of the National Academy of Sciences of the United States of America. 2013; 110(5): 1857-62.

Corrigan-Curay Jacqueline, Cohen-Haguenauer Odile, O'Reilly Marina, Ross Susan R, Fan Hung, Rosenberg Naomi, Somia Nikunj, King Nancy, Friedmann Theodore, Dunbar Cynthia, Aiuti Alessandro, Naldini Luigi, Baum Christopher, von Kalle Christof, Kiem Hans-Peter, Montini Eugenio, Bushman Frederic, Sorrentino Brian P, Carrondo Manuel, Malech Harry, Gahrton Gösta, Shapiro Robyn, Wolff Linda, Rosenthal Eugene, Jambou Robert, Zaia John, Kohn Donald B, Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy. Molecular therapy : the journal of the American Society of Gene Therapy. 2012; 20(6): 1084-94.

Karumbayaram Saravanan, Lee Peiyee, Azghadi Soheila F, Cooper Aaron R, Patterson Michaela, Kohn Donald B, Pyle April, Clark Amander, Byrne James, Zack Jerome A, Plath Kathrin, Lowry William E, From skin biopsy to neurons through a pluripotent intermediate under Good Manufacturing Practice protocols. Stem cells translational medicine. 2012; 1(1): 36-43.

Kohn Donald B, Pai Sung-Yun, Sadelain Michel, Gene Therapy by Autologous Transplant of Gene-Modified HSC. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2012; .

Candotti Fabio, Shaw Kit L, Muul Linda, Carbonaro Denise, Sokolic Robert, Choi Christopher, Schurman Shepherd H, Garabedian Elizabeth, Kesserwan Chimene, Jagadeesh G Jayashree, Fu Pei-Yu, Gschweng Eric, Cooper Aaron, Tisdale John F, Weinberg Kenneth I, Crooks Gay M, Kapoor Neena, Shah Ami, Abdel-Azim Hisham, Yu Xiao-Jin, Smogorzewska Monika, Wayne Alan S, Rosenblatt Howard M, Davis Carla M, Hanson Celine, Rishi Radha G, Wang Xiaoyan, Gjertson David, Yang Otto O, Balamurugan Arumugam, Bauer Gerhard, Ireland Joanna A, Engel Barbara C, Podsakoff Gregory M, Hershfield Michael S, Blaese R Michael, Parkman Robertson, Kohn Donald B, Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. Blood. 2012; .

Carbonaro Denise A, Jin Xiangyang, Wang Xingchao, Yu Xiao-Jin, Rozengurt Nora, Kaufman Michael L, Wang Xiaoyan, Gjertson David, Zhou Yang, Blackburn Michael R, Kohn Donald B, Gene therapy/bone marrow transplant in ADA-deficient mice: roles of enzyme replacement therapy and cytoreduction. Blood. 2012; .

Tarantal Alice F, Giannoni Francesca, Lee C Chang I, Wherley Jennifer, Sumiyoshi Teiko, Martinez Michele, Kahl Christoph A, Elashoff David, Louie Stan G, Kohn Donald B, Nonmyeloablative conditioning regimen to increase engraftment of gene-modified hematopoietic stem cells in young rhesus monkeys. Molecular therapy : the journal of the American Society of Gene Therapy. 2012; 20(5): 1033-45.

Parekh Chintan, Sahaghian Arineh, Kim William, Scholes Jessica, Ge Shundi, Zhu Yuhua, Asgharzadeh Shahab, Hollis Roger, Kohn Donald, Ji Lingyun, Malvar Jemily, Wang Xiaoyan, Crooks Gay, Novel pathways to erythropoiesis induced by dimerization of intracellular C-Mpl in human hematopoietic progenitors. Stem cells (Dayton, Ohio). 2012; 30(4): 697-708.

Shaw Kit L, Kohn Donald B, A Tale of Two SCIDs. Science Translational Medicine. 2011; 3(97): 97ps36.

Vatakis Dimitrios N, Koya Richard C, Nixon Christopher C, Wei Liu, Kim Sohn G, Avancena Patricia, Bristol Gregory, Baltimore David, Kohn Donald B, Ribas Antoni, Radu Caius G, Galic Zoran, Zack Jerome A, Antitumor activity from antigen-specific CD8 T cells generated in vivo from genetically engineered human hematopoietic stem cells. Proceedings of the National Academy of Sciences of the United States of America. 2011; 108(51): E1408-16.

Kohn Donald B, Dotti Gianpietro, Brentjens Renier, Savoldo Barbara, Jensen Michael, Cooper Laurence Jn, June Carl H, Rosenberg Steven, Sadelain Michel, Heslop Helen E, CARs on track in the clinic. Molecular therapy : the journal of the American Society of Gene Therapy. 2011; 19(3): 432-8.

Horwitz Edwin M, Horowitz Mary M, DiFronzo Nancy L, Kohn Donald B, Heslop Helen E, Heslop Helen E, Guidance for developing phase II cell therapy trial proposals for consideration by the Blood and Marrow Transplant Clinical Trials Network. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2011; 17(2): 192-6.

Cooper Aaron R, Patel Sanjeet, Senadheera Shantha, Plath Kathrin, Kohn Donald B, Hollis Roger P, Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration. Journal of virological methods. 2011; 177(1): 1-9.

Sokolic Robert, Maric Irina, Kesserwan Chimene, Garabedian Elizabeth, Hanson I Celine, Dodds Margaret, Buckley Rebecca, Issekutz Andrew C, Kamani Naynesh, Shaw Kit, Tan Ben, Bali Pawan, Hershfield Michael S, Kohn Donald B, Wayne Alan S, Candotti Fabio, Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency. Blood. 2011; 118(10): 2688-94.

Holt Nathalia, Wang Jianbin, Kim Kenneth, Friedman Geoffrey, Wang Xingchao, Taupin Vanessa, Crooks Gay M, Kohn Donald B, Gregory Philip D, Holmes Michael C, Cannon Paula M, Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nature biotechnology. 2010; 28(8): 839-47.

Shah Ami J, Kohn Donald B, Neurocognitive function of patients with severe combined immunodeficiency. Immunology and allergy clinics of North America. 2010; 30(1): 143-51.

Becker P S, Taylor J A, Trobridge G D, Zhao X, Beard B C, Chien S, Adair J, Kohn D B, Wagner J E, Shimamura A, Kiem H-P, Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector. Gene therapy. 2010; 17(10): 1244-52.

Pais Eszter, Park Jean, Alexy Tamas, Nikolian Vahagn, Ge Shundi, Shaw Kit, Senadheera Shantha, Hardee Cinnamon L, Skelton Dianne, Hollis Roger, Crooks Gay M, Kohn Donald B, Regulated Expansion of Human Pancreatic beta-Cells. Molecular therapy : the journal of the American Society of Gene Therapy. 2010; 135(2): .

Kohn Donald B, Update on gene therapy for immunodeficiencies. Clinical immunology (Orlando, Fla.). 2010; 135(2): 247-54.

Gruber Tanja A, Shah Ami J, Hernandez Michelle, Crooks Gay M, Abdel-Azim Hisham, Gupta Sudhir, McKnight Sean, White Drew, Kapoor Neena, Kohn Donald B, Clinical and genetic heterogeneity in Omenn syndrome and severe combined immune deficiency. Pediatric transplantation. 2009; 13(2): 244-50.

Kohn Donald B, Candotti Fabio, Gene therapy fulfilling its promise. The New England journal of medicine. 2009; 360(5): 518-21.

Griffith Linda M, Cowan Morton J, Notarangelo Luigi D, Puck Jennifer M, Buckley Rebecca H, Candotti Fabio, Conley Mary Ellen, Fleisher Thomas A, Gaspar H Bobby, Kohn Donald B, Ochs Hans D, O'Reilly Richard J, Rizzo J Douglas, Roifman Chaim M, Small Trudy N, Shearer William T, Shearer William T, Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management. The Journal of allergy and clinical immunology. 2009; 124(6): 1152-60.e12.

Shaw K L, Pais E, Ge S, Hardee C, Skelton D, Hollis R P, Crooks G M, Kohn D B, Lentiviral vectors with amplified beta cell-specific gene expression. Gene therapy. 2009; 16(8): 998-1008.

Lin Malinda, Epport Karen, Azen Colleen, Parkman Robertson, Kohn Donald B, Shah Ami J, Long-term neurocognitive function of pediatric patients with severe combined immune deficiency (SCID): pre- and post-hematopoietic stem cell transplant (HSCT). Journal of clinical immunology. 2009; 29(2): 231-7.

Shah Ami J, Kapoor Neena, Cooper Robert M, Crooks Gay M, Lenarsky Carl, Abdel-Azim Hisham, Wu Shi Qi, Wilson Kathy, Weinberg Kenneth I, Parkman Robertson, Kohn Donald B, Pre- and post-natal treatment of hemophagocytic lymphohistiocytosis. Pediatric blood & cancer. 2009; 52(1): 139-42.

Sondergaard Claus S, Haldrup Christa, Beer Christiane, Andersen Bente, Kohn Donald B, Pedersen Lene, Preloading potential of retroviral vectors is packaging cell clone dependent and centrifugation onto CH-296 ensures highest transduction efficiency. Human gene therapy. 2009; 20(4): 337-49.

Sumiyoshi Teiko, Holt Nathalia G, Hollis Roger P, Ge Shundi, Cannon Paula M, Crooks Gay M, Kohn Donald B, Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system. Human gene therapy. 2009; 20(12): 1607-26.

Griffith Linda M, Cowan Morton J, Kohn Donald B, Notarangelo Luigi D, Puck Jennifer M, Schultz Kirk R, Buckley Rebecca H, Eapen Mary, Kamani Naynesh R, O'Reilly Richard J, Parkman Robertson, Roifman Chaim M, Sullivan Kathleen E, Filipovich Alexandra H, Fleisher Thomas A, Shearer William T, Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs. The Journal of allergy and clinical immunology. 2008; 122(6): 1087-96.

Chhabra Arvind, Yang Lili, Wang Pin, Comin-Anduix Begoña, Das Raja, Chakraborty Nitya G, Ray Swagatam, Mehrotra Shikhar, Yang Haiguang, Hardee Cinnamon L, Hollis Roger, Dorsky David I, Koya Richard, Kohn Donald B, Ribas Antoni, Economou James S, Baltimore David, Mukherji Bijay, CD4+CD25- T cells transduced to express MHC class I-restricted epitope-specific TCR synthesize Th1 cytokines and exhibit MHC class I-restricted cytolytic effector function in a human melanoma model. Journal of immunology (Baltimore, Md. : 1950). 2008; 181(2): 1063-70.

Abdel-Azim Hisham, Zhu Yuhua, Hollis Roger, Wang Xiuli, Ge Shundi, Hao Qian-Lin, Smbatyan Goar, Kohn Donald B, Rosol Michael, Crooks Gay M, Expansion of multipotent and lymphoid-committed human progenitors through intracellular dimerization of Mpl. Blood. 2008; 111(8): 4064-74.

Taylor Jason A, Vojtech Lucia, Bahner Ingrid, Kohn Donald B, Laer Dorothee Von, Russell David W, Richard Robert E, Foamy virus vectors expressing anti-HIV transgenes efficiently block HIV-1 replication. Molecular therapy : the journal of the American Society of Gene Therapy. 2008; 16(1): 46-51.

Kohn D B, Gene therapy for childhood immunological diseases. Bone marrow transplantation. 2008; 41(2): 199-205.

Bonde Jesper, Wirthlin Louisa, Kohn Donald B, Nolta Jan A, Human hematopoietic cell culture, transduction, and analyses. Current protocols in human genetics / editorial board, Jonathan L. Haines ... [et al.]. 2008; Chapter 13(7): Unit 13.7.

Bauer Gerhard, Dao Mo A, Case Scott S, Meyerrose Todd, Wirthlin Louisa, Zhou Ping, Wang Xiuli, Herrbrich Phillip, Arevalo Jesusa, Csik Susie, Skelton Dianne C, Walker Jon, Pepper Karen, Kohn Donald B, Nolta Jan A, In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors. Molecular therapy : the journal of the American Society of Gene Therapy. 2008; 16(7): 1308-15.

Carbonaro Denise A, Jin Xiangyang, Cotoi Daniel, Mi Tiejuan, Yu Xiao-Jin, Skelton Dianne C, Dorey Frederick, Kellems Rodney E, Blackburn Michael R, Kohn Donald B, Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion. Blood. 2008; 111(12): 5745-54.

Shah Ami J, Epport Karen, Azen Colleen, Killen Renna, Wilson Kathy, De Clerck Dominique, Crooks Gay, Kapoor Neena, Kohn Donald B, Parkman Robertson, Weinberg Kenneth I, Progressive declines in neurocognitive function among survivors of hematopoietic stem cell transplantation for pediatric hematologic malignancies. Journal of pediatric hematology/oncology : official journal of the American Society of Pediatric Hematology/Oncology. 2008; 30(6): 411-8.

Singh Harjeet, Manuri Pallavi R, Olivares Simon, Dara Navid, Dawson Margaret J, Huls Helen, Hackett Perry B, Kohn Donald B, Shpall Elizabeth J, Champlin Richard E, Cooper Laurence J N, Redirecting specificity of T-cell populations for CD19 using the Sleeping Beauty system. Cancer research. 2008; 68(8): 2961-71.

Kahl C A, Cannon P M, Oldenburg J, Tarantal A F, Kohn D B, Tissue-specific restriction of cyclophilin A-independent HIV-1- and SIV-derived lentiviral vectors. Gene therapy. 2008; 15(15): 1079-89.

Anilkumar Gopalakrishnapillai, Barwe Sonali P, Christiansen Jason J, Rajasekaran Sigrid A, Kohn Donald B, Rajasekaran Ayyappan K, Association of prostate-specific membrane antigen with caveolin-1 and its caveolae-dependent internalization in microvascular endothelial cells: implications for targeting to tumor vasculature. Microvascular research. 2007; 72(1-2): 54-61.

Blumenthal Martina, Skelton Dianne, Pepper Karen A, Jahn Thomas, Methangkool Emily, Kohn Donald B, Effective suicide gene therapy for leukemia in a model of insertional oncogenesis in mice. Molecular therapy : the journal of the American Society of Gene Therapy. 2007; 15(1): 183-92.

Fischer-Lougheed J Y, Tarantal A F, Shulkin I, Mitsuhashi N, Kohn D B, Lee C C I, Kearns-Jonker M, Gene therapy to inhibit xenoantibody production using lentiviral vectors in non-human primates. Gene therapy. 2007; 14(1): 49-57.

Rossi John J, June Carl H, Kohn Donald B, Genetic therapies against HIV. Nature biotechnology. 2007; 25(12): 1444-54.

Bahner Ingrid, Sumiyoshi Teiko, Kagoda Mercy, Swartout Robin, Peterson Denise, Pepper Karen, Dorey Fred, Reiser Jacob, Kohn Donald B, Lentiviral vector transduction of a dominant-negative Rev gene into human CD34+ hematopoietic progenitor cells potently inhibits human immunodeficiency virus-1 replication. Molecular therapy : the journal of the American Society of Gene Therapy. 2007; 15(1): 76-85.

Kohn Donald B, Lentiviral vectors ready for prime-time. Nature biotechnology. 2007; 25(1): 65-6.

Rodriguez Ryan T, Velkey J Matthew, Lutzko Carolyn, Seerke Rina, Kohn Donald B, O'Shea K Sue, Firpo Meri T, Manipulation of OCT4 levels in human embryonic stem cells results in induction of differential cell types. Experimental biology and medicine (Maywood, N.J.). 2007; 232(10): 1368-80.

Barwe Sonali P, Maul Raymond S, Christiansen Jason J, Anilkumar Gopalakrishnapillai, Cooper Carlton R, Kohn Donald B, Rajasekaran Ayyappan K, Preferential association of prostate cancer cells expressing prostate specific membrane antigen to bone marrow matrix. International journal of oncology. 2007; 30(4): 899-904.

Engel Barbara C, Podsakoff Greg M, Ireland Joanna L, Smogorzewska E Monika, Carbonaro Denise A, Wilson Kathy, Shah Ami, Kapoor Neena, Sweeney Mirna, Borchert Mark, Crooks Gay M, Weinberg Kenneth I, Parkman Robertson, Rosenblatt Howard M, Wu Shi-Qi, Hershfield Michael S, Candotti Fabio, Kohn Donald B, Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report. Blood. 2007; 109(2): 503-6.

Shah Ami J, Kapoor Neena, Crooks Gay M, Weinberg Kenneth I, Azim Hisham Abdel, Killen Renna, Kuo Lily, Rushing Teresa, Kohn Donald B, Parkman Robertson, The effects of Campath 1H upon graft-versus-host disease, infection, relapse, and immune reconstitution in recipients of pediatric unrelated transplants. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2007; 13(5): 584-93.

Higashimoto T, Urbinati F, Perumbeti A, Jiang G, Zarzuela A, Chang L-J, Kohn D B, Malik P, The woodchuck hepatitis virus post-transcriptional regulatory element reduces readthrough transcription from retroviral vectors. Gene therapy. 2007; 14(17): 1298-304.

Kahl Christoph A, Tarantal Alice F, Lee Chang I, Jimenez Daniel F, Choi Christopher, Pepper Karen, Petersen Denise, Fletcher Misty D, Leapley Alyssa C, Fisher Jennifer, Burns Travis S, Ultsch Man-Ni, Dorey Frederick J, Kohn Donald B, Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector. Experimental hematology. 2006; 34(3): 369-81.

Carbonaro Denise A, Jin Xiangyang, Petersen Denise, Wang Xingchao, Dorey Fred, Kil Ki Soo, Aldrich Melissa, Blackburn Michael R, Kellems Rodney E, Kohn Donald B, In vivo transduction by intravenous injection of a lentiviral vector expressing human ADA into neonatal ADA gene knockout mice: a novel form of enzyme replacement therapy for ADA deficiency. Molecular therapy : the journal of the American Society of Gene Therapy. 2006; 13(6): 1110-20.

Unwalla Hoshang J, Li Hai-Tang, Bahner Ingrid, Li Ming-Jie, Kohn Donald, Rossi John J, Novel Pol II fusion promoter directs human immunodeficiency virus type 1-inducible coexpression of a short hairpin RNA and protein. Journal of virology. 2006; 80(4): 1863-73.

Jang Jung-Eun, Shaw Kit, Yu Xiao-Jin, Petersen Denise, Pepper Karen, Lutzko Carolyn, Kohn Donald B, Specific and stable gene transfer to human embryonic stem cells using pseudotyped lentiviral vectors. Stem cells and development. 2006; 15(1): 109-17.

Hollis Roger P, Nightingale Sarah J, Wang Xiuli, Pepper Karen A, Yu Xiao-Jin, Barsky Lora, Crooks Gay M, Kohn Donald B, Stable gene transfer to human CD34(+) hematopoietic cells using the Sleeping Beauty transposon. Experimental hematology. 2006; 34(10): 1333-43.

Mitsuhashi Noboru, Fischer-Lougheed Jacqueline, Shulkin Irina, Kleihauer Annette, Kohn Donald B, Weinberg Kenneth I, Starnes Vaughn A, Kearns-Jonker Mary, Tolerance induction by lentiviral gene therapy with a nonmyeloablative regimen. Blood. 2006; 107(6): 2286-93.

Nightingale Sarah J, Hollis Roger P, Pepper Karen A, Petersen Denise, Yu Xiao-Jin, Yang Catherine, Bahner Ingrid, Kohn Donald B, Transient gene expression by nonintegrating lentiviral vectors. Molecular therapy : the journal of the American Society of Gene Therapy. 2006; 13(6): 1121-32.

Lee C Chang I, Jimenez Daniel F, Kohn Donald B, Tarantal Alice F, Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta). Human gene therapy. 2005; 16(4): 417-25.

Jimenez D F, Lee C I, O'Shea C E, Kohn D B, Tarantal A F, HIV-1-derived lentiviral vectors and fetal route of administration on transgene biodistribution and expression in rhesus monkeys. Gene therapy. 2005; 12(10): 821-30.

Tarantal Alice F, McDonald Ruth J, Jimenez Daniel F, Lee C Chang I, O'Shea Cristin E, Leapley Alyssa C, Won Rosa H, Plopper Charles G, Lutzko Carolyn, Kohn Donald B, Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery. Molecular therapy : the journal of the American Society of Gene Therapy. 2005; 12(1): 87-98.

Chan Belinda, Wara Diane, Bastian John, Hershfield Michael S, Bohnsack John, Azen Colleen G, Parkman Robertson, Weinberg Kenneth, Kohn Donald B, Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID). Clinical immunology (Orlando, Fla.). 2005; 117(2): 133-43.

Kobayashi Hiroshi, Carbonaro Denise, Pepper Karen, Petersen Denise, Ge Shundi, Jackson Holly, Shimada Hiroyuki, Moats Rex, Kohn Donald B, Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector. Molecular therapy : the journal of the American Society of Gene Therapy. 2005; 11(5): 776-89.

Podsakoff Greg M, Engel Barbara C, Kohn Donald B, Perspectives on gene therapy for immune deficiencies. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2005; 11(12): 972-6.

Gruber Tanja A, Skelton Dianne C, Kohn Donald B, Recombinant murine interleukin-12 elicits potent antileukemic immune responses in a murine model of Philadelphia chromosome-positive acute lymphoblastic leukemia. Cancer gene therapy. 2005; 12(10): 818-24.

Podsakoff Greg M, Engel Barbara C, Carbonaro Denise A, Choi Chris, Smogorzewska Elzbieta M, Bauer Gerhard, Selander David, Csik Susan, Wilson Kathy, Betts Michael R, Koup Richard A, Nabel Gary J, Bishop Keith, King Steven, Schmidt Manfred, von Kalle Christof, Church Joseph A, Kohn Donald B, Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells. Molecular therapy : the journal of the American Society of Gene Therapy. 2005; 12(1): 77-86.

Shah Ami J, Kapoor Neena, Crooks Gay M, Parkman Robertson, Weinberg Kenneth I, Wilson Kathy, Kohn Donald B, Successful hematopoietic stem cell transplantation for Niemann-Pick disease type B. Pediatrics. 2005; 116(4): 1022-5.

Shah Ami J, Lenarsky Carl, Kapoor Neena, Crooks Gay M, Kohn Donald B, Parkman Robertson, Epport Karen, Wilson Kathy, Weinberg Ken, Busulfan and cyclophosphamide as a conditioning regimen for pediatric acute lymphoblastic leukemia patients undergoing bone marrow transplantation. Journal of pediatric hematology/oncology. 2004; 26(2): 91-7.

Logan Aaron C, Nightingale Sarah J, Haas Dennis L, Cho Gerald J, Pepper Karen A, Kohn Donald B, Factors influencing the titer and infectivity of lentiviral vectors. Human gene therapy. 2004; 15(10): 976-88.

Logan Aaron C, Haas Dennis L, Kafri Tal, Kohn Donald B, Integrated self-inactivating lentiviral vectors produce full-length genomic transcripts competent for encapsidation and integration. Journal of virology. 2004; 78(16): 8421-36.

Lee Chang I, Kohn Donald B, Ekert Jason E, Tarantal Alice F, Morphological analysis and lentiviral transduction of fetal monkey bone marrow-derived mesenchymal stem cells. Molecular therapy : the journal of the American Society of Gene Therapy. 2004; 9(1): 112-23.

Zheng Yi, Ryazantsev Sergey, Ohmi Kazuhiro, Zhao Hui-Zhi, Rozengurt Nora, Kohn Donald B, Neufeld Elizabeth F, Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB. Molecular genetics and metabolism. 2004; 82(4): 286-95.

Lee Chang I, Cowan Morton J, Kohn Donald B, Tarantal Alice F, Simian immunodeficiency virus infection of hematopoietic stem cells and bone marrow stromal cells. Journal of acquired immune deficiency syndromes (1999). 2004; 36(1): 553-61.

Kearns-Jonker Mary, Fischer-Lougheed Jacqueline, Shulkin Irina, Kleihauer Annette, Mitsuhashi Noboru, Kohn Donald B, Weinberg Kenneth, D'Apice Anthony J F, Starnes Vaughn A, Cramer Donald V, Use of lentiviral vectors to induce long-term tolerance to gal(+) heart grafts. Transplantation. 2004; 77(11): 1748-54.

Kohn Donald B, Sadelain Michel, Dunbar Cynthia, Bodine David, Kiem Hans-Peter, Candotti Fabio, Tisdale John, Riviére Isabelle, Blau C Anthony, Richard Robert E, Sorrentino Brian, Nolta Jan, Malech Harry, Brenner Malcolm, Cornetta Kenneth, Cavagnaro Joy, High Katherine, Glorioso Joseph, Glorioso Joseph, American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Molecular therapy : the journal of the American Society of Gene Therapy. 2003; 8(2): 180-7.

Schmidt Manfred, Carbonaro Denise A, Speckmann Carsten, Wissler Manuela, Bohnsack John, Elder Melissa, Aronow Bruce J, Nolta Jan A, Kohn Donald B, von Kalle Christof, Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates. Nature medicine. 2003; 9(4): 463-8.

Wang Xiuli, Rosol Michael, Ge Shundi, Peterson Denise, McNamara George, Pollack Harvey, Kohn Donald B, Nelson Marvin D, Crooks Gay M, Dynamic tracking of human hematopoietic stem cell engraftment using in vivo bioluminescence imaging. Blood. 2003; 102(10): 3478-82.

Wang Lijun, Haas Dennis, Halene Stephanie, Kohn Donald B, Effects of the negative control region on expression from retroviral LTR. Molecular therapy : the journal of the American Society of Gene Therapy. 2003; 7(4): 438-40.

Schmidt Manfred, Glimm Hanno, Wissler Manuela, Hoffmann Gesa, Olsson Karin, Sellers Stephanie, Carbonaro Denise, Tisdale John F, Leurs Cordula, Hanenberg Helmut, Dunbar Cynthia E, Kiem Hans-Peter, Karlsson Stefan, Kohn Donald B, Williams David, Von Kalle Christof, Efficient characterization of retro-, lenti-, and foamyvector-transduced cell populations by high-accuracy insertion site sequencing. Annals of the New York Academy of Sciences. 2003; 996(7): 112-21.

Engel Barbara C, Kohn Donald B, Gene therapy for inborn and acquired immune deficiency disorders. Acta haematologica. 2003; 110(2-3): 60-70.

Lutzko Carolyn, Senadheera Dinithi, Skelton Dianne, Petersen Denise, Kohn Donald B, Lentivirus vectors incorporating the immunoglobulin heavy chain enhancer and matrix attachment regions provide position-independent expression in B lymphocytes. Journal of virology. 2003; 77(13): 7341-51.

Kohn Donald B, Gänsbacher Bernd, Letter to the editors of Nature from the American Society of Gene Therapy (ASGT) and the European Society of Gene Therapy (ESGT). The journal of gene medicine. 2003; 5(7): 641.

Kohn Donald B, Sadelain Michel, Glorioso Joseph C, Occurrence of leukaemia following gene therapy of X-linked SCID. Nature reviews. Cancer. 2003; 3(7): 477-88.

Kurre Peter, Morris Julia, Thomasson Bobbie, Kohn Donald B, Kiem Hans-Peter, Scaffold attachment region-containing retrovirus vectors improve long-term proviral expression after transplantation of GFP-modified CD34+ baboon repopulating cells. Blood. 2003; 102(9): 3117-9.

Haas Dennis L, Lutzko Carolyn, Logan Aaron C, Cho Gerald J, Skelton Dianne, Jin Yu Xiao, Pepper Karen A, Kohn Donald B, The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells. Journal of virology. 2003; 77(17): 9439-50.

Burgos J S, Rosol M, Moats R A, Khankaldyyan V, Kohn D B, Nelson M D, Laug W E, Time course of bioluminescent signal in orthotopic and heterotopic brain tumors in nude mice. BioTechniques. 2003; 34(6): 1184-8.

Goto Hiroaki, Yang Bo, Petersen Denise, Pepper Karen A, Alfaro Paul A, Kohn Donald B, Reynolds C Patrick, Transduction of green fluorescent protein increased oxidative stress and enhanced sensitivity to cytotoxic drugs in neuroblastoma cell lines. Molecular cancer therapeutics. 2003; 2(9): 911-7.

Zheng Yi, Rozengurt Nora, Ryazantsev Sergey, Kohn Donald B, Satake Noriko, Neufeld Elizabeth F, Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow. Molecular genetics and metabolism. 2003; 79(4): 233-44.

Engel Barbara C, Kohn Donald B, Podsakoff Greg M, Update on gene therapy of inherited immune deficiencies. Current opinion in molecular therapeutics. 2003; 5(5): 503-7.

Smogorzewska Elzbieta Monika, Weinberg Kenneth I, Kohn Donald B, [Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments]. Medycyna wieku rozwojowego. 2003; 7(1): 27-34.

Kohn Donald B, Adenosine deaminase gene therapy protocol revisited. Molecular therapy : the journal of the American Society of Gene Therapy. 2002; 5(2): 96-7.

Logan Aaron C, Lutzko Carolyn, Kohn Donald B, Advances in lentiviral vector design for gene-modification of hematopoietic stem cells. Current opinion in biotechnology. 2002; 13(5): 429-36.

O'Rourke J P, Newbound G C, Kohn D B, Olsen J C, Bunnell B A, Comparison of gene transfer efficiencies and gene expression levels achieved with equine infectious anemia virus- and human immunodeficiency virus type 1-derived lentivirus vectors. Journal of virology. 2002; 76(3): 1510-5.

Price Mary A, Case Scott S, Carbonaro Denise A, Yu Xiao-Jin, Petersen Denise, Sabo Kathleen M, Curran Michael A, Engel Barbara C, Margarian Hovanes, Abkowitz Janis L, Nolan Garry P, Kohn Donald B, Crooks Gay M, Expression from second-generation feline immunodeficiency virus vectors is impaired in human hematopoietic cells. Molecular therapy : the journal of the American Society of Gene Therapy. 2002; 6(5): 645-52.

Yates Frank, Malassis-Séris Michèle, Stockholm Daniel, Bouneaud Cécile, Larousserie Frédérique, Noguiez-Hellin Patricia, Danos Olivier, Kohn Donald B, Fischer Alain, de Villartay Jean-Pierre, Cavazzana-Calvo Marina, Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency. Blood. 2002; 100(12): 3942-9.

Meertens Lisa, Zhao Yongjun, Rosic-Kablar Suzana, Li Liheng, Chan Kin, Dobson Howard, Gartley Cathy, Lutzko Carolyn, Hopwood John, Kohn Donald, Kruth Stephen, Hough Margaret R, Dubé Ian D, In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression. Human gene therapy. 2002; 13(15): 1809-20.

Kurre Peter, Morris Julia, Andrews Robert G, Kohn Donald B, Kiem Hans-Peter, Kinetics of fluorescence expression in nonhuman primates transplanted with GFP retrovirus-modified CD34 cells. Molecular therapy : the journal of the American Society of Gene Therapy. 2002; 6(1): 83-90.

Grove Joanna E, Lutzko Carolyn, Priller Josef, Henegariu Octavian, Theise Neil D, Kohn Donald B, Krause Diane S, Marrow-derived cells as vehicles for delivery of gene therapy to pulmonary epithelium. American journal of respiratory cell and molecular biology. 2002; 27(6): 645-51.

Gruber Tanja A, Skelton Dianne C, Kohn Donald B, Requirement for NK cells in CD40 ligand-mediated rejection of Philadelphia chromosome-positive acute lymphoblastic leukemia cells. Journal of immunology (Baltimore, Md. : 1950). 2002; 168(1): 73-80.

Shah Ami J, Kapoor Neena, Weinberg Kenneth I, Crooks Gay M, Kohn Donald B, Lenarsky Carl, Kaufman Francine, Epport Karen, Wilson Kathy, Parkman Robertson, Second hematopoietic stem cell transplantation in pediatric patients: overall survival and long-term follow-up. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2002; 8(4): 221-8.

Kohn D B, Gene therapy for genetic haematological disorders and immunodeficiencies. Journal of internal medicine. 2001; 249(4): 379-90.

Weinberg K I, Kapoor N, Shah A J, Crooks G M, Kohn D B, Parkman R, Hematopoietic stem cell transplantation for severe combined immune deficiency. Current allergy and asthma reports. 2001; 1(5): 416-20.

Tarantal A F, Lee C I, Ekert J E, McDonald R, Kohn D B, Plopper C G, Case S S, Bunnell B A, Lentiviral vector gene transfer into fetal rhesus monkeys (Macaca mulatta): lung-targeting approaches. Molecular therapy : the journal of the American Society of Gene Therapy. 2001; 4(6): 614-21.

Tarantal A F, O'Rourke J P, Case S S, Newbound G C, Li J, Lee C I, Baskin C R, Kohn D B, Bunnell B A, Rhesus monkey model for fetal gene transfer: studies with retroviral- based vector systems. Molecular therapy : the journal of the American Society of Gene Therapy. 2001; 3(2): 128-38.

Skelton D, Satake N, Kohn D B, The enhanced green fluorescent protein (eGFP) is minimally immunogenic in C57BL/6 mice. Gene therapy. 2001; 8(23): 1813-4.

Kohn D B, Gene therapy for XSCID: the first success of gene therapy. Pediatric research. 2000; 48(5): 578.

Bauer G, Selander D, Engel B, Carbonaro D, Csik S, Rawlings S, Church J, Kohn D B, Gene therapy for pediatric AIDS. Annals of the New York Academy of Sciences. 2000; 918(2): 318-29.