PubRank

Donald B Kohn

Author PubWeight™ 110.99‹?›

Top papers

Rank Title Journal Year PubWeight™‹?›
1 Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol 2010 6.55
2 Redirecting specificity of T-cell populations for CD19 using the Sleeping Beauty system. Cancer Res 2008 2.57
3 Genetic therapies against HIV. Nat Biotechnol 2007 2.50
4 Transient gene expression by nonintegrating lentiviral vectors. Mol Ther 2006 1.89
5 Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. Blood 2012 1.85
6 Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med 2014 1.81
7 Dynamic tracking of human hematopoietic stem cell engraftment using in vivo bioluminescence imaging. Blood 2003 1.79
8 Marrow-derived cells as vehicles for delivery of gene therapy to pulmonary epithelium. Am J Respir Cell Mol Biol 2002 1.70
9 Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: results of the first 2 years. J Allergy Clin Immunol 2013 1.66
10 Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates. Nat Med 2003 1.65
11 Antitumor activity from antigen-specific CD8 T cells generated in vivo from genetically engineered human hematopoietic stem cells. Proc Natl Acad Sci U S A 2011 1.61
12 Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Hum Gene Ther 2013 1.54
13 Expansion of multipotent and lymphoid-committed human progenitors through intracellular dimerization of Mpl. Blood 2008 1.51
14 A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med 2014 1.42
15 Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID). Clin Immunol 2005 1.40
16 The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consortium prospective study 6901. J Clin Immunol 2013 1.40
17 Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency. J Clin Invest 2017 1.39
18 Prostate cancer originating in basal cells progresses to adenocarcinoma propagated by luminal-like cells. Proc Natl Acad Sci U S A 2013 1.37
19 Factors influencing the titer and infectivity of lentiviral vectors. Hum Gene Ther 2004 1.32
20 American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Mol Ther 2003 1.29
21 Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells. Mol Ther 2005 1.28
22 Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs. J Allergy Clin Immunol 2008 1.25
23 Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector. Mol Ther 2005 1.25
24 Gene therapy fulfilling its promise. N Engl J Med 2009 1.24
25 Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management. J Allergy Clin Immunol 2009 1.23
26 β-globin gene transfer to human bone marrow for sickle cell disease. J Clin Invest 2013 1.22
27 The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells. J Virol 2003 1.17
28 Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow. Mol Genet Metab 2003 1.15
29 Generation and characterization of transgene-free human induced pluripotent stem cells and conversion to putative clinical-grade status. Stem Cell Res Ther 2013 1.14
30 Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction. Blood 2012 1.14
31 In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors. Mol Ther 2008 1.14
32 Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery. Mol Ther 2005 1.14
33 Advances in lentiviral vector design for gene-modification of hematopoietic stem cells. Curr Opin Biotechnol 2002 1.13
34 Stable gene transfer to human CD34(+) hematopoietic cells using the Sleeping Beauty transposon. Exp Hematol 2006 1.13
35 Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus. Mol Ther 2013 1.08
36 Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system. Hum Gene Ther 2009 1.08
37 Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB. Mol Genet Metab 2004 1.08
38 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. Mol Ther 2006 1.07
39 From skin biopsy to neurons through a pluripotent intermediate under Good Manufacturing Practice protocols. Stem Cells Transl Med 2011 1.07
40 Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration. J Virol Methods 2011 1.06
41 Integrated self-inactivating lentiviral vectors produce full-length genomic transcripts competent for encapsidation and integration. J Virol 2004 1.06
42 Transduction of green fluorescent protein increased oxidative stress and enhanced sensitivity to cytotoxic drugs in neuroblastoma cell lines. Mol Cancer Ther 2003 1.03
43 Morphological analysis and lentiviral transduction of fetal monkey bone marrow-derived mesenchymal stem cells. Mol Ther 2004 1.01
44 Adoptive transfer of MART-1 T-cell receptor transgenic lymphocytes and dendritic cell vaccination in patients with metastatic melanoma. Clin Cancer Res 2014 1.01
45 Manipulation of OCT4 levels in human embryonic stem cells results in induction of differential cell types. Exp Biol Med (Maywood) 2007 1.00
46 Modification of hematopoietic stem/progenitor cells with CD19-specific chimeric antigen receptors as a novel approach for cancer immunotherapy. Hum Gene Ther 2013 1.00
47 Allelic exclusion and peripheral reconstitution by TCR transgenic T cells arising from transduced human hematopoietic stem/progenitor cells. Mol Ther 2013 1.00
48 Effective suicide gene therapy for leukemia in a model of insertional oncogenesis in mice. Mol Ther 2007 0.99
49 Progressive declines in neurocognitive function among survivors of hematopoietic stem cell transplantation for pediatric hematologic malignancies. J Pediatr Hematol Oncol 2008 0.99
50 Lentivirus vectors incorporating the immunoglobulin heavy chain enhancer and matrix attachment regions provide position-independent expression in B lymphocytes. J Virol 2003 0.98
51 Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy. Mol Ther 2012 0.98
52 Lentiviral vectors ready for prime-time. Nat Biotechnol 2007 0.97
53 Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector. Exp Hematol 2006 0.97
54 Long-term in vivo monitoring of mouse and human hematopoietic stem cell engraftment with a human positron emission tomography reporter gene. Proc Natl Acad Sci U S A 2013 0.96
55 Efficient characterization of retro-, lenti-, and foamyvector-transduced cell populations by high-accuracy insertion site sequencing. Ann N Y Acad Sci 2003 0.96
56 Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis. Mol Ther 2012 0.96
57 Hematopoietic stem cells for cancer immunotherapy. Immunol Rev 2014 0.95
58 Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report. Blood 2006 0.95
59 Lentiviral vector transduction of a dominant-negative Rev gene into human CD34+ hematopoietic progenitor cells potently inhibits human immunodeficiency virus-1 replication. Mol Ther 2007 0.94
60 Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta). Hum Gene Ther 2005 0.93
61 Specific and stable gene transfer to human embryonic stem cells using pseudotyped lentiviral vectors. Stem Cells Dev 2006 0.92
62 A tale of two SCIDs. Sci Transl Med 2011 0.91
63 Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency. Blood 2011 0.90
64 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 0.90
65 Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency. Blood 2002 0.90
66 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. J Immunol 2008 0.90
67 Scaffold attachment region-containing retrovirus vectors improve long-term proviral expression after transplantation of GFP-modified CD34+ baboon repopulating cells. Blood 2003 0.89
68 The effects of Campath 1H upon graft-versus-host disease, infection, relapse, and immune reconstitution in recipients of pediatric unrelated transplants. Biol Blood Marrow Transplant 2007 0.88
69 Clinical and genetic heterogeneity in Omenn syndrome and severe combined immune deficiency. Pediatr Transplant 2008 0.87
70 Foamy virus vectors expressing anti-HIV transgenes efficiently block HIV-1 replication. Mol Ther 2007 0.86
71 Long-term neurocognitive function of pediatric patients with severe combined immune deficiency (SCID): pre- and post-hematopoietic stem cell transplant (HSCT). J Clin Immunol 2008 0.86
72 Expression from second-generation feline immunodeficiency virus vectors is impaired in human hematopoietic cells. Mol Ther 2002 0.86
73 Association of prostate-specific membrane antigen with caveolin-1 and its caveolae-dependent internalization in microvascular endothelial cells: implications for targeting to tumor vasculature. Microvasc Res 2006 0.85
74 Enrichment of human hematopoietic stem/progenitor cells facilitates transduction for stem cell gene therapy. Stem Cells 2015 0.85
75 Preferential association of prostate cancer cells expressing prostate specific membrane antigen to bone marrow matrix. Int J Oncol 2007 0.85
76 Busulfan and cyclophosphamide as a conditioning regimen for pediatric acute lymphoblastic leukemia patients undergoing bone marrow transplantation. J Pediatr Hematol Oncol 2004 0.85
77 Successful hematopoietic stem cell transplantation for Niemann-Pick disease type B. Pediatrics 2005 0.84
78 Kinetics of fluorescence expression in nonhuman primates transplanted with GFP retrovirus-modified CD34 cells. Mol Ther 2002 0.83
79 Nonmyeloablative conditioning regimen to increase engraftment of gene-modified hematopoietic stem cells in young rhesus monkeys. Mol Ther 2012 0.83
80 Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013. Hum Gene Ther 2014 0.82
81 Use of lentiviral vectors to induce long-term tolerance to gal(+) heart grafts. Transplantation 2004 0.82
82 Simian immunodeficiency virus infection of hematopoietic stem cells and bone marrow stromal cells. J Acquir Immune Defic Syndr 2004 0.80
83 Erythropoiesis from human embryonic stem cells through erythropoietin-independent AKT signaling. Stem Cells 2014 0.79
84 Effects of the negative control region on expression from retroviral LTR. Mol Ther 2003 0.79
85 Dissecting the mechanism of histone deacetylase inhibitors to enhance the activity of zinc finger nucleases delivered by integrase-defective lentiviral vectors. Hum Gene Ther 2014 0.79
86 Update on gene therapy of inherited immune deficiencies. Curr Opin Mol Ther 2003 0.78
87 Gene therapy for inborn and acquired immune deficiency disorders. Acta Haematol 2003 0.78
88 Requirement for NK cells in CD40 ligand-mediated rejection of Philadelphia chromosome-positive acute lymphoblastic leukemia cells. J Immunol 2002 0.78
89 Preloading potential of retroviral vectors is packaging cell clone dependent and centrifugation onto CH-296 ensures highest transduction efficiency. Hum Gene Ther 2009 0.78
90 Neurocognitive function of patients with severe combined immunodeficiency. Immunol Allergy Clin North Am 2010 0.76
91 Recombinant murine interleukin-12 elicits potent antileukemic immune responses in a murine model of Philadelphia chromosome-positive acute lymphoblastic leukemia. Cancer Gene Ther 2005 0.76
92 Guidance for developing phase II cell therapy trial proposals for consideration by the Blood and Marrow Transplant Clinical Trials Network. Biol Blood Marrow Transplant 2010 0.76
93 Tolerance induction by lentiviral gene therapy with a nonmyeloablative regimen. Blood 2005 0.76
94 Perspectives on gene therapy for immune deficiencies. Biol Blood Marrow Transplant 2005 0.76
95 A CD19/Fc fusion protein for detection of anti-CD19 chimeric antigen receptors. J Transl Med 2013 0.75
96 Pre- and post-natal treatment of hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer 2009 0.75
97 Regulated expansion of human pancreatic beta-cells. Mol Ther 2010 0.75
98 Letter to the editors of Nature from the American Society of Gene Therapy (ASGT) and the European Society of Gene Therapy (ESGT). J Gene Med 2003 0.75
99 Human hematopoietic cell culture, transduction, and analyses. Curr Protoc Hum Genet 2008 0.75
100 [Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments]. Med Wieku Rozwoj 2003 0.75
101 Second hematopoietic stem cell transplantation in pediatric patients: overall survival and long-term follow-up. Biol Blood Marrow Transplant 2002 0.75