PubRank

Luigi Naldini

Author PubWeight™ 218.74‹?›

Top papers

Rank Title Journal Year PubWeight™‹?›
1 Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol 2007 9.23
2 Tie2 identifies a hematopoietic lineage of proangiogenic monocytes required for tumor vessel formation and a mesenchymal population of pericyte progenitors. Cancer Cell 2005 7.90
3 An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat Biotechnol 2011 6.44
4 Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 2013 5.64
5 Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol 2006 5.58
6 Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat Biotechnol 2007 5.36
7 Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 2013 5.34
8 Targeting exogenous genes to tumor angiogenesis by transplantation of genetically modified hematopoietic stem cells. Nat Med 2003 4.02
9 Targeting the ANG2/TIE2 axis inhibits tumor growth and metastasis by impairing angiogenesis and disabling rebounds of proangiogenic myeloid cells. Cancer Cell 2011 4.02
10 FcRgamma activation regulates inflammation-associated squamous carcinogenesis. Cancer Cell 2010 3.80
11 Stable knockdown of microRNA in vivo by lentiviral vectors. Nat Methods 2008 3.55
12 A distinguishing gene signature shared by tumor-infiltrating Tie2-expressing monocytes, blood "resident" monocytes, and embryonic macrophages suggests common functions and developmental relationships. Blood 2009 3.52
13 The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. J Clin Invest 2009 3.34
14 Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer. Nat Med 2006 3.22
15 Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications. Nat Rev Genet 2009 3.09
16 Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters. Nat Biotechnol 2004 2.78
17 Identification of proangiogenic TIE2-expressing monocytes (TEMs) in human peripheral blood and cancer. Blood 2007 2.74
18 Forebrain ependymal cells are Notch-dependent and generate neuroblasts and astrocytes after stroke. Nat Neurosci 2009 2.74
19 Cleavage of the plasma membrane Na+/Ca2+ exchanger in excitotoxicity. Cell 2005 2.63
20 Targeting the tumor and its microenvironment by a dual-function decoy Met receptor. Cancer Cell 2004 2.61
21 Site-specific integration and tailoring of cassette design for sustainable gene transfer. Nat Methods 2011 2.30
22 A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. Blood 2007 2.28
23 Comprehensive genomic access to vector integration in clinical gene therapy. Nat Med 2009 2.19
24 Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy. Blood 2009 1.88
25 Tie2-expressing monocytes: regulation of tumor angiogenesis and therapeutic implications. Trends Immunol 2007 1.84
26 Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Sci Transl Med 2010 1.84
27 Generation of HIV-1 derived lentiviral vectors. Methods Enzymol 2002 1.81
28 Tumor-targeted interferon-alpha delivery by Tie2-expressing monocytes inhibits tumor growth and metastasis. Cancer Cell 2008 1.80
29 Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection. Blood 2011 1.76
30 Systemic and targeted delivery of semaphorin 3A inhibits tumor angiogenesis and progression in mouse tumor models. Arterioscler Thromb Vasc Biol 2011 1.75
31 Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo. Blood 2002 1.71
32 Tie2-expressing monocytes and tumor angiogenesis: regulation by hypoxia and angiopoietin-2. Cancer Res 2007 1.70
33 Elusive identities and overlapping phenotypes of proangiogenic myeloid cells in tumors. Am J Pathol 2010 1.70
34 In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance. Blood 2007 1.69
35 Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk. Hepatology 2011 1.66
36 TIE2-expressing macrophages limit the therapeutic efficacy of the vascular-disrupting agent combretastatin A4 phosphate in mice. J Clin Invest 2011 1.66
37 Generation of potent and stable human CD4+ T regulatory cells by activation-independent expression of FOXP3. Mol Ther 2007 1.64
38 A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR. Blood 2012 1.61
39 Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells. J Clin Invest 2004 1.59
40 Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice. Blood 2003 1.56
41 Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. J Clin Invest 2006 1.54
42 Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells. Blood 2004 1.53
43 Human T lymphocytes transduced by lentiviral vectors in the absence of TCR activation maintain an intact immune competence. Blood 2003 1.50
44 Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice. Blood 2012 1.46
45 Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors. Mol Ther 2002 1.41
46 Robust in vivo gene transfer into adult mammalian neural stem cells by lentiviral vectors. Proc Natl Acad Sci U S A 2004 1.40
47 HIV-based vectors. Preparation and use. Methods Mol Med 2002 1.39
48 Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application. Hum Gene Ther 2011 1.37
49 The MET oncogene drives a genetic programme linking cancer to haemostasis. Nature 2005 1.37
50 The first reported generation of several induced pluripotent stem cell lines from homozygous and heterozygous Huntington's disease patients demonstrates mutation related enhanced lysosomal activity. Neurobiol Dis 2012 1.35
51 Attenuation of miR-126 activity expands HSC in vivo without exhaustion. Cell Stem Cell 2012 1.35
52 CD44v6-targeted T cells mediate potent antitumor effects against acute myeloid leukemia and multiple myeloma. Blood 2013 1.34
53 In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance. Blood 2009 1.34
54 Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. Blood 2010 1.33
55 Role of haematopoietic cells and endothelial progenitors in tumour angiogenesis. Biochim Biophys Acta 2006 1.30
56 miR-511-3p modulates genetic programs of tumor-associated macrophages. Cell Rep 2012 1.29
57 Regulated and multiple miRNA and siRNA delivery into primary cells by a lentiviral platform. Mol Ther 2009 1.25
58 An uncleavable form of pro-scatter factor suppresses tumor growth and dissemination in mice. J Clin Invest 2004 1.25
59 In vivo targeting of tumor endothelial cells by systemic delivery of lentiviral vectors. Hum Gene Ther 2003 1.22
60 Neural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy. Stem Cells 2011 1.22
61 Stability of lentiviral vector-mediated transgene expression in the brain in the presence of systemic antivector immune responses. Hum Gene Ther 2005 1.20
62 Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells. Mol Ther 2002 1.18
63 Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome. Mol Ther 2012 1.18
64 Proteasome activity restricts lentiviral gene transfer into hematopoietic stem cells and is down-regulated by cytokines that enhance transduction. Blood 2006 1.16
65 A role for miR-155 in enabling tumor-infiltrating innate immune cells to mount effective antitumor responses in mice. Blood 2013 1.16
66 Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models. Mol Ther 2009 1.15
67 Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation. Proc Natl Acad Sci U S A 2012 1.12
68 Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector. Hum Gene Ther 2005 1.12
69 A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A. Mol Ther 2011 1.11
70 Reprogramming T lymphocytes for melanoma adoptive immunotherapy by T-cell receptor gene transfer with lentiviral vectors. Cancer Res 2009 1.10
71 Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations. J Clin Invest 2012 1.10
72 Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination. Mol Ther 2013 1.10
73 MET overexpression turns human primary osteoblasts into osteosarcomas. Cancer Res 2006 1.09
74 A human immunodeficiency virus type 1 pol gene-derived sequence (cPPT/CTS) increases the efficiency of transduction of human nondividing monocytes and T lymphocytes by lentiviral vectors. Hum Gene Ther 2002 1.08
75 Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer. Nat Methods 2013 1.08
76 Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I. Mol Ther 2010 1.08
77 miR-142-3p prevents macrophage differentiation during cancer-induced myelopoiesis. Immunity 2013 1.07
78 Transduction of a gene expression cassette using advanced generation lentiviral vectors. Methods Enzymol 2002 1.07
79 Efficient Tet-dependent expression of human factor IX in vivo by a new self-regulating lentiviral vector. Mol Ther 2005 1.07
80 Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction. Mol Ther 2004 1.06
81 Lentiviral vector integration profiles differ in rodent postmitotic tissues. Mol Ther 2011 1.06
82 Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Nonobese diabetic/severe combined immunodeficient. Blood 2002 1.06
83 Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects. Blood 2006 1.04
84 Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation. Hum Gene Ther 2006 1.03
85 Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome. J Allergy Clin Immunol 2011 1.03
86 SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies. Biochem J 2007 1.02
87 Angiopoietin-2 TIEs up macrophages in tumor angiogenesis. Clin Cancer Res 2011 1.02
88 Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models. Hum Mol Genet 2010 1.02
89 Genetic engineering of hematopoiesis for targeted IFN-α delivery inhibits breast cancer progression. Sci Transl Med 2014 1.01
90 TIE2-expressing monocytes/macrophages regulate revascularization of the ischemic limb. EMBO Mol Med 2013 0.99
91 Quantitative proteomic analysis of lentiviral vectors using 2-DE. Proteomics 2009 0.98
92 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
93 RNAi technology and lentiviral delivery as a powerful tool to suppress Tpr-Met-mediated tumorigenesis. Cancer Gene Ther 2005 0.97
94 Transcriptional targeting of lentiviral vectors by long terminal repeat enhancer replacement. J Virol 2002 0.96
95 Tracking differentiating neural progenitors in pluripotent cultures using microRNA-regulated lentiviral vectors. Proc Natl Acad Sci U S A 2010 0.96
96 Tie2-expressing monocytes (TEMs): novel targets and vehicles of anticancer therapy? Biochim Biophys Acta 2009 0.95
97 Oncoretroviral and lentiviral vector-mediated gene therapy. Methods Enzymol 2002 0.95
98 Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery. Mol Ther 2005 0.94
99 The immune response to lentiviral-delivered transgene is modulated in vivo by transgene-expressing antigen-presenting cells but not by CD4+CD25+ regulatory T cells. Blood 2007 0.93
100 Lentiviral vector gene transfer is limited by the proteasome at postentry steps in various types of stem cells. Stem Cells 2008 0.93
101 Gene therapy of storage disorders by retroviral and lentiviral vectors. Hum Gene Ther 2005 0.91
102 Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice. EMBO Mol Med 2013 0.91
103 Forkhead box protein 3 (FOXP3) mutations lead to increased TH17 cell numbers and regulatory T-cell instability. J Allergy Clin Immunol 2011 0.91
104 Short-term culture of human CD34+ cells for lentiviral gene transfer. Methods Mol Biol 2009 0.90
105 A microRNA-based system for selecting and maintaining the pluripotent state in human induced pluripotent stem cells. Stem Cells 2011 0.89
106 Efficiency of onco-retroviral and lentiviral gene transfer into primary mouse and human B-lymphocytes is pseudotype dependent. Hum Gene Ther 2003 0.89
107 Treatment of the mouse model of mucopolysaccharidosis type IIIB with lentiviral-NAGLU vector. Biochem J 2005 0.88
108 Deletion in a (T)8 microsatellite abrogates expression regulation by 3'-UTR. Nucleic Acids Res 2003 0.88
109 HIV-1-derived lentiviral vectors directly activate plasmacytoid dendritic cells, which in turn induce the maturation of myeloid dendritic cells. Hum Gene Ther 2011 0.88
110 Lentiviral gene transfer ameliorates disease progression in Long-Evans cinnamon rats: an animal model for Wilson disease. Scand J Gastroenterol 2006 0.87
111 Fighting rare diseases: the model of the telethon research institutes in Italy. Hum Gene Ther 2015 0.86
112 CD4⁺ T cells from IPEX patients convert into functional and stable regulatory T cells by FOXP3 gene transfer. Sci Transl Med 2013 0.86
113 Retroviral vectors containing Tet-controlled bidirectional transcription units for simultaneous regulation of two gene activities. J Mol Genet Med 2006 0.85
114 Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting. Mol Ther 2011 0.84
115 Monitoring disease evolution and treatment response in lysosomal disorders by the peripheral benzodiazepine receptor ligand PK11195. Neurobiol Dis 2009 0.83
116 Correction of mucopolysaccharidosis type IIIb fibroblasts by lentiviral vector-mediated gene transfer. Biochem J 2002 0.83
117 A double-switch vector system positively regulates transgene expression by endogenous microRNA expression (miR-ON vector). Mol Ther 2013 0.83
118 Manipulating Immune Tolerance with Micro-RNA Regulated Gene Therapy. Front Microbiol 2011 0.83
119 Development and maturation of invariant NKT cells in the presence of lysosomal engulfment. Eur J Immunol 2009 0.83
120 The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cell niche. Blood 2010 0.83
121 Effects of phosphorylation and neuronal activity on the control of synapse formation by synapsin I. J Cell Sci 2011 0.82
122 Dynamic Activity of miR-125b and miR-93 during Murine Neural Stem Cell Differentiation In Vitro and in the Subventricular Zone Neurogenic Niche. PLoS One 2013 0.80
123 miR-126 Regulates Distinct Self-Renewal Outcomes in Normal and Malignant Hematopoietic Stem Cells. Cancer Cell 2016 0.80
124 Characterization of new arylsulfatase A gene mutations reinforces genotype-phenotype correlation in metachromatic leukodystrophy. Hum Mutat 2009 0.80
125 Inserting optimism into gene therapy. Nat Med 2006 0.79
126 Immune responses in liver-directed lentiviral gene therapy. Transl Res 2013 0.79
127 In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectors. Eur J Biochem 2002 0.79
128 Novel candidate disease for gene therapy: metachromatic leukodystrophy. Expert Opin Biol Ther 2007 0.78
129 Cellular innate immunity and restriction of viral infection: implications for lentiviral gene therapy in human hematopoietic cells. Hum Gene Ther 2015 0.77
130 Lentiviral transduction of primary myeloma cells with CD80 and CD154 generates antimyeloma effector T cells. Hum Gene Ther 2005 0.77
131 Antagonizing metastasis. Nat Biotechnol 2010 0.77
132 Good news on the clinical gene transfer front. Hum Gene Ther 2008 0.75
133 Corrigendum to "Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott-Aldrich Syndrome in Preclinical Models". Mol Ther 2016 0.75