Published in Curr Gene Ther on December 01, 2009
Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies. J Clin Invest (2010) 2.13
Integration-free iPS cells engineered using human artificial chromosome vectors. PLoS One (2011) 1.13
siRNA Versus miRNA as Therapeutics for Gene Silencing. Mol Ther Nucleic Acids (2015) 1.12
Prion-like nanofibrils of small molecules (PriSM) selectively inhibit cancer cells by impeding cytoskeleton dynamics. J Biol Chem (2014) 0.96
Retinal dystrophies and gene therapy. Eur J Pediatr (2011) 0.94
Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients. Hum Gene Ther (2012) 0.90
Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A. Gene Ther (2012) 0.86
Reversal of diabetes through gene therapy of diabetic rats by hepatic insulin expression via lentiviral transduction. Mol Ther (2012) 0.84
Let There Be Light: Gene and Cell Therapy for Blindness. Hum Gene Ther (2016) 0.79
International Society for the Advancement of Cytometry cell sorter biosafety standards. Cytometry A (2014) 0.79
Virally mediated gene manipulation in the adult CNS. Front Mol Neurosci (2011) 0.79
Therapeutic and prophylactic applications of bacteriophage components in modern medicine. Cold Spring Harb Perspect Med (2014) 0.78
General considerations on the biosafety of virus-derived vectors used in gene therapy and vaccination. Curr Gene Ther (2013) 0.78
Retroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectives. J Transl Med (2016) 0.78
Development of an equine-tropic replication-competent lentivirus assay for equine infectious anemia virus-based lentiviral vectors. Hum Gene Ther Methods (2012) 0.77
The determination of importance of sequences neighboring the Psi sequence in lentiviral vector transduction and packaging efficiency. PLoS One (2012) 0.77
Shedding of clinical-grade lentiviral vectors is not detected in a gene therapy setting. Gene Ther (2015) 0.76
Tracking stem cells with superparamagnetic iron oxide nanoparticles: perspectives and considerations. Int J Nanomedicine (2017) 0.76
Sustained delivery of siRNA/PEI complex from in situ forming hydrogels potently inhibits the proliferation of gastric cancer. J Exp Clin Cancer Res (2016) 0.75
Adenovirally delivered enzyme prodrug therapy with herpes simplex virus-thymidine kinase in composite tissue free flaps shows therapeutic efficacy in rat models of glioma. Plast Reconstr Surg (2015) 0.75
HIV-1 integrase forms stable tetramers and associates with LEDGF/p75 protein in human cells. J Biol Chem (2002) 4.91
Clinical presentation and penetrance of pheochromocytoma/paraganglioma syndromes. J Clin Endocrinol Metab (2005) 4.07
LEDGF/p75 is essential for nuclear and chromosomal targeting of HIV-1 integrase in human cells. J Biol Chem (2003) 3.97
A differentiation checkpoint limits hematopoietic stem cell self-renewal in response to DNA damage. Cell (2012) 3.21
Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood (2006) 3.07
Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain. Hum Gene Ther (2007) 2.64
Physiological promoters reduce the genotoxic risk of integrating gene vectors. Mol Ther (2008) 2.56
Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors. Mol Ther (2009) 2.56
Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Hum Gene Ther (2010) 2.51
Rational design of small-molecule inhibitors of the LEDGF/p75-integrase interaction and HIV replication. Nat Chem Biol (2010) 2.48
Integrase mutants defective for interaction with LEDGF/p75 are impaired in chromosome tethering and HIV-1 replication. J Biol Chem (2005) 2.44
Transportin-SR2 imports HIV into the nucleus. Curr Biol (2008) 2.35
The interaction of LEDGF/p75 with integrase is lentivirus-specific and promotes DNA binding. J Biol Chem (2005) 2.28
The succinate dehydrogenase genetic testing in a large prospective series of patients with paragangliomas. J Clin Endocrinol Metab (2009) 2.22
CD4⁺ follicular helper T cell infiltration predicts breast cancer survival. J Clin Invest (2013) 2.18
Transient and stable knockdown of the integrase cofactor LEDGF/p75 reveals its role in the replication cycle of human immunodeficiency virus. J Virol (2006) 2.09
Overcoming reprogramming resistance of Fanconi anemia cells. Blood (2012) 2.07
Long-term reversal of diabetes in non-obese diabetic mice by liver-directed gene therapy. J Gene Med (2013) 2.02
Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity. Sci Transl Med (2014) 2.00
The phenotype of human STK4 deficiency. Blood (2012) 1.84
Overexpression of the lens epithelium-derived growth factor/p75 integrase binding domain inhibits human immunodeficiency virus replication. J Virol (2006) 1.82
LEDGF hybrids efficiently retarget lentiviral integration into heterochromatin. Mol Ther (2010) 1.72
Differential effects of progenitor cell populations on left ventricular remodeling and myocardial neovascularization after myocardial infarction. J Am Coll Cardiol (2010) 1.70
Lentiviral vector design and imaging approaches to visualize the early stages of cellular reprogramming. Mol Ther (2011) 1.69
Cellular co-factors of HIV-1 integration. Trends Biochem Sci (2006) 1.68
NAMPT is essential for the G-CSF-induced myeloid differentiation via a NAD(+)-sirtuin-1-dependent pathway. Nat Med (2009) 1.66
European position paper on the anatomical terminology of the internal nose and paranasal sinuses. Rhinol Suppl (2014) 1.63
Impact of the central polypurine tract on the kinetics of human immunodeficiency virus type 1 vector transduction. J Virol (2003) 1.61
Pharmacological targeting of the thrombomodulin-activated protein C pathway mitigates radiation toxicity. Nat Med (2012) 1.58
Gene therapy: X-SCID transgene leukaemogenicity. Nature (2006) 1.57
T-cell receptor gene transfer exclusively to human CD8(+) cells enhances tumor cell killing. Blood (2012) 1.55
Hepatic lentiviral gene transfer is associated with clonal selection, but not with tumor formation in serially transplanted rodents. Hepatology (2013) 1.55
Highly efficient multicistronic lentiviral vectors with peptide 2A sequences. Hum Gene Ther (2009) 1.54
Development of resistance against diketo derivatives of human immunodeficiency virus type 1 by progressive accumulation of integrase mutations. J Virol (2003) 1.54
LEF-1 is crucial for neutrophil granulocytopoiesis and its expression is severely reduced in congenital neutropenia. Nat Med (2006) 1.52
Characterization of lentiviral vector-mediated gene transfer in adult mouse brain. Hum Gene Ther (2002) 1.50
Factors associated with the mediastinal spread of cervical necrotizing fasciitis. Ann Thorac Surg (2011) 1.48
Identification and characterization of a functional nuclear localization signal in the HIV-1 integrase interactor LEDGF/p75. J Biol Chem (2004) 1.47
Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. Mol Ther (2008) 1.44
Comparison of lentiviral vector titration methods. BMC Biotechnol (2006) 1.42
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med (2014) 1.42
Virus evolution reveals an exclusive role for LEDGF/p75 in chromosomal tethering of HIV. PLoS Pathog (2007) 1.41
Small-molecule inhibitors of the LEDGF/p75 binding site of integrase block HIV replication and modulate integrase multimerization. Antimicrob Agents Chemother (2012) 1.40
Loss-of-function mutations in the IL-21 receptor gene cause a primary immunodeficiency syndrome. J Exp Med (2013) 1.39
Transcriptional enhancers induce insertional gene deregulation independently from the vector type and design. Mol Ther (2009) 1.39
Upscaling of lentiviral vector production by tangential flow filtration. J Gene Med (2005) 1.38
Role of nitrogen in transmucosal gas exchange rate in the rat middle ear. J Appl Physiol (1985) (2006) 1.38
MN1 overexpression induces acute myeloid leukemia in mice and predicts ATRA resistance in patients with AML. Blood (2007) 1.37
Bromo- and extraterminal domain chromatin regulators serve as cofactors for murine leukemia virus integration. J Virol (2013) 1.34
Genetic modification through oligonucleotide-mediated mutagenesis. A GMO regulatory challenge? Environ Biosafety Res (2009) 1.33
Lens epithelium-derived growth factor/p75 interacts with the transposase-derived DDE domain of PogZ. J Biol Chem (2009) 1.30
Protein transduction from retroviral Gag precursors. Proc Natl Acad Sci U S A (2010) 1.30
Multiple mutations in human immunodeficiency virus-1 integrase confer resistance to the clinical trial drug S-1360. AIDS (2004) 1.28
Mice with ribosomal protein S19 deficiency develop bone marrow failure and symptoms like patients with Diamond-Blackfan anemia. Blood (2011) 1.28
The BET family of proteins targets moloney murine leukemia virus integration near transcription start sites. Cell Rep (2013) 1.26
LEDGF/p75-independent HIV-1 replication demonstrates a role for HRP-2 and remains sensitive to inhibition by LEDGINs. PLoS Pathog (2012) 1.25
Identification of the LEDGF/p75 binding site in HIV-1 integrase. J Mol Biol (2006) 1.24
Engineering nucleases for gene targeting: safety and regulatory considerations. N Biotechnol (2013) 1.24
Preparation of bioactive soluble human leukemia inhibitory factor from recombinant Escherichia coli using thioredoxin as fusion partner. Protein Expr Purif (2010) 1.22
Lentiviral nuclear import: a complex interplay between virus and host. Bioessays (2007) 1.22
Rescue of ATP7B function in hepatocyte-like cells from Wilson's disease induced pluripotent stem cells using gene therapy or the chaperone drug curcumin. Hum Mol Genet (2011) 1.20
LEDGINs inhibit late stage HIV-1 replication by modulating integrase multimerization in the virions. Retrovirology (2013) 1.17
Interplay between HIV entry and transportin-SR2 dependency. Retrovirology (2011) 1.17
Discovery of novel non-cytotoxic salicylhydrazide containing HIV-1 integrase inhibitors. Bioorg Med Chem Lett (2007) 1.16
Novel targets for HIV therapy. Antiviral Res (2008) 1.15
Fetal surgery is a clinical reality. Semin Fetal Neonatal Med (2009) 1.15
Differential interaction of HIV-1 integrase and JPO2 with the C terminus of LEDGF/p75. J Mol Biol (2007) 1.14
New class of HIV integrase inhibitors that block viral replication in cell culture. Curr Biol (2002) 1.13
Intracranial complications of rhinosinusitis. A review, typical imaging data and algorithm of management. Rhinology (2009) 1.12
Noninvasive monitoring of long-term lentiviral vector-mediated gene expression in rodent brain with bioluminescence imaging. Mol Ther (2006) 1.11
Depletion of PINK1 affects mitochondrial metabolism, calcium homeostasis and energy maintenance. J Cell Sci (2011) 1.11
Lentiviral vector-mediated delivery of short hairpin RNA results in persistent knockdown of gene expression in mouse brain. Hum Gene Ther (2003) 1.11
Tumor cells escape suicide gene therapy by genetic and epigenetic instability. Blood (2004) 1.11
miR-99a/100~125b tricistrons regulate hematopoietic stem and progenitor cell homeostasis by shifting the balance between TGFβ and Wnt signaling. Genes Dev (2014) 1.11
Design of a novel cyclotide-based CXCR4 antagonist with anti-human immunodeficiency virus (HIV)-1 activity. J Med Chem (2012) 1.10
Quantitative evaluation of MRI-based tracking of ferritin-labeled endogenous neural stem cell progeny in rodent brain. Neuroimage (2012) 1.10
Mutations in human immunodeficiency virus type 1 integrase confer resistance to the naphthyridine L-870,810 and cross-resistance to the clinical trial drug GS-9137. Antimicrob Agents Chemother (2008) 1.10
Gene therapy on the move. EMBO Mol Med (2013) 1.10
The LEDGF/p75 integrase interaction, a novel target for anti-HIV therapy. Virology (2013) 1.10