Published in Gene Ther on December 03, 2009
In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature (2011) 4.81
Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial. Hum Gene Ther (2011) 2.29
Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood (2013) 2.09
Hypothalamic-specific manipulation of Fto, the ortholog of the human obesity gene FTO, affects food intake in rats. PLoS One (2010) 1.89
Telomerase gene therapy in adult and old mice delays aging and increases longevity without increasing cancer. EMBO Mol Med (2012) 1.66
LRH-1-dependent glucose sensing determines intermediary metabolism in liver. J Clin Invest (2012) 1.58
Overcoming preexisting humoral immunity to AAV using capsid decoys. Sci Transl Med (2013) 1.56
Intraganglionic AAV6 results in efficient and long-term gene transfer to peripheral sensory nervous system in adult rats. PLoS One (2013) 1.51
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy. J Clin Invest (2013) 1.44
Capsid-expressing DNA in AAV vectors and its elimination by use of an oversize capsid gene for vector production. Gene Ther (2010) 1.29
Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome. J Neurosci (2013) 1.26
Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver. Gene Ther (2010) 1.21
Enhancing muscle membrane repair by gene delivery of MG53 ameliorates muscular dystrophy and heart failure in δ-Sarcoglycan-deficient hamsters. Mol Ther (2012) 1.13
Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B. Mol Ther (2012) 1.13
Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain. Nat Biotechnol (2016) 1.11
Vascular endothelial growth factor-mediated islet hypervascularization and inflammation contribute to progressive reduction of β-cell mass. Diabetes (2012) 1.11
Robust ZFN-mediated genome editing in adult hemophilic mice. Blood (2013) 1.11
Empty Virions In AAV8 Vector Preparations Reduce Transduction Efficiency And May Cause Total Viral Particle Dose-Limiting Side-Effects. Mol Ther Methods Clin Dev (2014) 1.08
In vivo genome editing of the albumin locus as a platform for protein replacement therapy. Blood (2015) 1.03
Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors. Mol Ther (2015) 1.02
Transgene expression in target-defined neuron populations mediated by retrograde infection with adeno-associated viral vectors. J Neurosci (2013) 1.02
Identification of the heparin binding site on adeno-associated virus serotype 3B (AAV-3B). Virology (2011) 0.99
Rapid and simplified purification of recombinant adeno-associated virus. J Virol Methods (2012) 0.98
Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B. Front Microbiol (2011) 0.96
Analysis of particle content of recombinant adeno-associated virus serotype 8 vectors by ion-exchange chromatography. Hum Gene Ther Methods (2012) 0.94
AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease. Sci Transl Med (2015) 0.94
In vivo adeno-associated viral vector-mediated genetic engineering of white and brown adipose tissue in adult mice. Diabetes (2013) 0.93
Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material. Hum Gene Ther (2014) 0.91
Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice. Mol Ther (2011) 0.91
CD8+ T cell recognition of epitopes within the capsid of adeno-associated virus 8-based gene transfer vectors depends on vectors' genome. Mol Ther (2013) 0.91
Ex Vivo Expanded Autologous Polyclonal Regulatory T Cells Suppress Inhibitor Formation in Hemophilia. Mol Ther Methods Clin Dev (2014) 0.90
Single tyrosine mutation in AAV8 and AAV9 capsids is insufficient to enhance gene delivery to skeletal muscle and heart. Hum Gene Ther Methods (2012) 0.89
Safety study of adeno-associated virus serotype 2-mediated human acid sphingomyelinase expression in the nonhuman primate brain. Hum Gene Ther (2012) 0.89
Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy. Diabetes (2013) 0.88
AAV2/8 vectors purified from culture medium with a simple and rapid protocol transduce murine liver, muscle, and retina efficiently. Hum Gene Ther Methods (2013) 0.88
A simplified purification method for AAV variant by polyethylene glycol aqueous two-phase partitioning. Bioengineered (2012) 0.88
Proteomics analysis of co-purifying cellular proteins associated with rAAV vectors. PLoS One (2014) 0.87
AAV-mediated expression of an ADAMTS13 variant prevents shigatoxin-induced thrombotic thrombocytopenic purpura. Blood (2013) 0.87
A versatile adeno-associated virus vector producer cell line method for scalable vector production of different serotypes. Hum Gene Ther (2011) 0.86
Intracisternal delivery of AAV9 results in oligodendrocyte and motor neuron transduction in the whole central nervous system of cats. Gene Ther (2014) 0.85
Telomerase expression confers cardioprotection in the adult mouse heart after acute myocardial infarction. Nat Commun (2014) 0.85
3D Imaging of Axons in Transparent Spinal Cords from Rodents and Nonhuman Primates(,.) eNeuro (2015) 0.84
Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery. Mol Ther Methods Clin Dev (2014) 0.83
Adeno-associated virus-mediated overexpression of LARGE rescues α-dystroglycan function in dystrophic mice with mutations in the fukutin-related protein. Hum Gene Ther Methods (2014) 0.82
AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes. Mol Ther Methods Clin Dev (2015) 0.81
Quantification of AAV particle titers by infrared fluorescence scanning of coomassie-stained sodium dodecyl sulfate-polyacrylamide gels. Hum Gene Ther Methods (2012) 0.81
Muscle and heart function restoration in a limb girdle muscular dystrophy 2I (LGMD2I) mouse model by systemic FKRP gene delivery. Mol Ther (2014) 0.81
CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome). JCI Insight (2016) 0.81
Proinsulin slows retinal degeneration and vision loss in the P23H rat model of retinitis pigmentosa. Hum Gene Ther (2012) 0.81
Practical utilization of recombinant AAV vector reference standards: focus on vector genomes titration by free ITR qPCR. Mol Ther Methods Clin Dev (2016) 0.80
Human Glucocorticoid Receptor β Regulates Gluconeogenesis and Inflammation in Mouse Liver. Mol Cell Biol (2015) 0.79
K137R mutation on adeno-associated viral capsids had minimal effect on enhancing gene delivery in vivo. Hum Gene Ther Methods (2013) 0.79
AAV2 production with optimized N/P ratio and PEI-mediated transfection results in low toxicity and high titer for in vitro and in vivo applications. J Virol Methods (2013) 0.79
Use of self-complementary adeno-associated virus serotype 2 as a tracer for labeling axons: implications for axon regeneration. PLoS One (2014) 0.78
A new minimally-invasive method for microinjection into the mouse spinal dorsal horn. Sci Rep (2015) 0.77
The liver as a target organ for gene therapy: state of the art, challenges, and future perspectives. Pharmaceuticals (Basel) (2012) 0.77
Genome-wide RNAi screening identifies host restriction factors critical for in vivo AAV transduction. Proc Natl Acad Sci U S A (2015) 0.77
Systemic AAV9-IFNβ gene delivery treats highly invasive glioblastoma. Neuro Oncol (2016) 0.77
Advanced Characterization of DNA Molecules in rAAV Vector Preparations by Single-stranded Virus Next-generation Sequencing. Mol Ther Nucleic Acids (2015) 0.76
AAV8-mediated Sirt1 gene transfer to the liver prevents high carbohydrate diet-induced nonalcoholic fatty liver disease. Mol Ther Methods Clin Dev (2014) 0.76
Strategies for targeting primate neural circuits with viral vectors. J Neurophysiol (2016) 0.76
Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A. Mol Ther Methods Clin Dev (2016) 0.76
AAV-mediated Sirt1 overexpression in skeletal muscle activates oxidative capacity but does not prevent insulin resistance. Mol Ther Methods Clin Dev (2016) 0.75
Recombinant adeno-associated viral (rAAV) vectors mediate efficient gene transduction in cultured neonatal and adult microglia. J Neurochem (2015) 0.75
Adeno-Associated Viral Vectors Serotype 8 for Cell-Specific Delivery of Therapeutic Genes in the Central Nervous System. Front Neuroanat (2017) 0.75
Somatic genome editing with CRISPR/Cas9 generates and corrects a metabolic disease. Sci Rep (2017) 0.75
Impaired SUMOylation of nuclear receptor LRH-1 promotes nonalcoholic fatty liver disease. J Clin Invest (2017) 0.75
Loss of Frataxin activates the iron/sphingolipid/PDK1/Mef2 pathway in mammals. Elife (2016) 0.75
Scalable downstream strategies for purification of recombinant adeno- associated virus vectors in light of the properties. Curr Pharm Biotechnol (2015) 0.75
Overcoming Insulin Insufficiency by Forced Follistatin Expression in β-cells of db/db Mice. Mol Ther (2015) 0.75
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome. Mol Ther Methods Clin Dev (2016) 0.75
Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics. Mol Ther Nucleic Acids (2016) 0.75
A simplified system for generating recombinant adenoviruses. Proc Natl Acad Sci U S A (1998) 24.58
Requirement for p53 and p21 to sustain G2 arrest after DNA damage. Science (1998) 17.91
Extensive mosaic structure revealed by the complete genome sequence of uropathogenic Escherichia coli. Proc Natl Acad Sci U S A (2002) 13.83
Negative feedback regulation of TGF-beta signaling by the SnoN oncoprotein. Science (1999) 7.51
Blockade of NMDA receptors and apoptotic neurodegeneration in the developing brain. Science (1999) 6.37
Purification and biochemical heterogeneity of the mammalian SWI-SNF complex. EMBO J (1996) 6.36
Diversity and specialization of mammalian SWI/SNF complexes. Genes Dev (1996) 6.01
Human Smad3 and Smad4 are sequence-specific transcription activators. Mol Cell (1998) 5.88
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet (2000) 5.80
Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2. Nat Med (1999) 5.50
Complete genome sequence and comparative genomics of Shigella flexneri serotype 2a strain 2457T. Infect Immun (2003) 5.11
SOX9 directly regulates the type-II collagen gene. Nat Genet (1997) 4.88
Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nat Med (1999) 4.61
Post-transcriptional regulation of vascular endothelial growth factor mRNA by the product of the VHL tumor suppressor gene. Proc Natl Acad Sci U S A (1996) 3.88
Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc Natl Acad Sci U S A (1997) 3.84
Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet (1995) 3.74
Composite co-activator ARC mediates chromatin-directed transcriptional activation. Nature (1999) 3.32
Loss of P-glycoprotein expression in hematopoietic stem cells does not improve responses to imatinib in a murine model of chronic myelogenous leukemia. Leukemia (2005) 3.13
CIR, a corepressor linking the DNA binding factor CBF1 to the histone deacetylase complex. Proc Natl Acad Sci U S A (1999) 2.81
Identification of functionally variant MDR1 alleles among European Americans and African Americans. Clin Pharmacol Ther (2001) 2.79
Nucleotide sequence, transcription map, and mutation analysis of the 13q14 chromosomal region deleted in B-cell chronic lymphocytic leukemia. Blood (2001) 2.73
A specificity and targeting subunit of a human SWI/SNF family-related chromatin-remodeling complex. Mol Cell Biol (2000) 2.70
Critical role for alpha/beta and gamma interferons in persistence of lymphocytic choriomeningitis virus by clonal exhaustion of cytotoxic T cells. J Virol (2001) 2.69
The transcriptional cofactor complex CRSP is required for activity of the enhancer-binding protein Sp1. Nature (1999) 2.61
Conservation. Reconsidering the consequences of selective fisheries. Science (2012) 2.57
The Ski oncoprotein interacts with the Smad proteins to repress TGFbeta signaling. Genes Dev (1999) 2.52
RNA- and DNA-binding activities in hepatitis B virus capsid protein: a model for their roles in viral replication. J Virol (1992) 2.42
Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology (2009) 2.37
Architectural DNA binding by a high-mobility-group/kinesin-like subunit in mammalian SWI/SNF-related complexes. Proc Natl Acad Sci U S A (1998) 2.30
The chromo superfamily: new members, duplication of the chromo domain and possible role in delivering transcription regulators to chromatin. Nucleic Acids Res (1995) 2.26
Applicability of the International Index for aggressive lymphomas to patients with low-grade lymphoma. J Clin Oncol (1994) 2.20
Microsurgical replantation of the avulsed scalp: report of 20 cases. Plast Reconstr Surg (1996) 2.17
DNA array studies demonstrate convergent regulation of virulence factors by Cph1, Cph2, and Efg1 in Candida albicans. J Biol Chem (2001) 2.14
Abeta amyloid fibrils possess a core structure highly resistant to hydrogen exchange. Proc Natl Acad Sci U S A (2000) 2.02
Reconstitution of transcription factor SL1: exclusive binding of TBP by SL1 or TFIID subunits. Science (1994) 1.98
Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood (1998) 1.96
Human TAFII 105 is a cell type-specific TFIID subunit related to hTAFII130. Cell (1996) 1.96
Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation. Mol Ther (2001) 1.93
TATA box-binding protein (TBP)-related factor 2 (TRF2), a third member of the TBP family. Proc Natl Acad Sci U S A (1999) 1.86
Peripheral T-cell lymphomas: initial features, natural history, and prognostic factors in a series of 174 patients diagnosed according to the R.E.A.L. Classification. Ann Oncol (1998) 1.81
Renal failure in multiple myeloma: presenting features and predictors of outcome in 94 patients from a single institution. Arch Intern Med (1998) 1.79
Transient immunosuppression allows transgene expression following readministration of adeno-associated viral vectors. Hum Gene Ther (1998) 1.79
SREBP transcriptional activity is mediated through an interaction with the CREB-binding protein. Genes Dev (1996) 1.76
Alterations of striatal NMDA receptor subunits associated with the development of dyskinesia in the MPTP-lesioned primate model of Parkinson's disease. Neuropharmacology (2005) 1.75
Enteric bacterial catalysts for fuel ethanol production. Biotechnol Prog (1999) 1.74
A human condensin complex containing hCAP-C-hCAP-E and CNAP1, a homolog of Xenopus XCAP-D2, colocalizes with phosphorylated histone H3 during the early stage of mitotic chromosome condensation. Mol Cell Biol (2000) 1.73
VirB1, a component of the T-complex transfer machinery of Agrobacterium tumefaciens, is processed to a C-terminal secreted product, VirB1. J Bacteriol (1997) 1.72
Transgenic mice overexpressing phosphoenolpyruvate carboxykinase develop non-insulin-dependent diabetes mellitus. Proc Natl Acad Sci U S A (1994) 1.71
Canine hemophilia B resulting from a point mutation with unusual consequences. Proc Natl Acad Sci U S A (1989) 1.68
Identification of two distinct human SMC protein complexes involved in mitotic chromosome dynamics. Proc Natl Acad Sci U S A (1998) 1.68
Increased number of chromosomal imbalances and high-level DNA amplifications in mantle cell lymphoma are associated with blastoid variants. Blood (1999) 1.66
Exogenously regulated stem cell-mediated gene therapy for bone regeneration. Mol Ther (2001) 1.63
Survival after progression in patients with follicular lymphoma: analysis of prognostic factors. Ann Oncol (2002) 1.61
Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males. Mol Ther (2001) 1.61
A TRF1:BRF complex directs Drosophila RNA polymerase III transcription. Cell (2000) 1.59
Requirement for a kinase-specific chaperone pathway in the production of a Cdk9/cyclin T1 heterodimer responsible for P-TEFb-mediated tat stimulation of HIV-1 transcription. J Biol Chem (2000) 1.59
Major role of local immune responses in antibody formation to factor IX in AAV gene transfer. Gene Ther (2005) 1.58
Adeno-associated virus type 2-mediated gene transfer: role of cellular FKBP52 protein in transgene expression. J Virol (2001) 1.58
Engineering the metabolism of Escherichia coli W3110 for the conversion of sugar to redox-neutral and oxidized products: homoacetate production. Proc Natl Acad Sci U S A (2003) 1.57
Molecular cloning and expression of the 32-kDa subunit of human TFIID reveals interactions with VP16 and TFIIB that mediate transcriptional activation. Proc Natl Acad Sci U S A (1995) 1.57
Identification and characterization of a TFIID-like multiprotein complex from Saccharomyces cerevisiae. Proc Natl Acad Sci U S A (1995) 1.56
X-ray crystal structure of rabbit N-acetylglucosaminyltransferase I: catalytic mechanism and a new protein superfamily. EMBO J (2000) 1.56
Efficiency of eight different AAV serotypes in transducing rat myocardium in vivo. Gene Ther (2007) 1.56
A micromolar pool of antigenically distinct precursors is required to initiate cooperative assembly of hepatitis B virus capsids in Xenopus oocytes. J Virol (1993) 1.55
Abnormal compartmentalization of cartilage matrix components in mice lacking collagen X: implications for function. J Cell Biol (1997) 1.55
Successful expression of human factor IX following repeat administration of adenoviral vector in mice. Proc Natl Acad Sci U S A (1996) 1.55
Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX. Mol Ther (2000) 1.55
Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9. Mol Ther (2001) 1.54
The basic helix-loop-helix transcription factor Cph2 regulates hyphal development in Candida albicans partly via TEC1. Mol Cell Biol (2001) 1.52
Post-transcriptional modification in archaeal tRNAs: identities and phylogenetic relations of nucleotides from mesophilic and hyperthermophilic Methanococcales. Nucleic Acids Res (2001) 1.52
Transgenic mice overexpressing insulin-like growth factor-II in beta cells develop type 2 diabetes. J Clin Invest (2000) 1.51
Systematic review of chimney and periscope grafts for endovascular aneurysm repair. Br J Surg (2013) 1.50
A mutation in the propeptide of Factor IX leads to warfarin sensitivity by a novel mechanism. J Clin Invest (1996) 1.49
Haemophilia B: database of point mutations and short additions and deletions--fourth edition, 1993. Nucleic Acids Res (1993) 1.49
The Escherichia coli GTPase CgtAE cofractionates with the 50S ribosomal subunit and interacts with SpoT, a ppGpp synthetase/hydrolase. J Bacteriol (2004) 1.48
Nerve sprouting and sympathetic hyperinnervation in a canine model of atrial fibrillation produced by prolonged right atrial pacing. Circulation (2001) 1.45
Bone marrow assessment in B-cell chronic lymphocytic leukaemia: aspirate or biopsy? A comparative study in 258 patients. Br J Haematol (1996) 1.44
NOTCH1 mutations identify a genetic subgroup of chronic lymphocytic leukemia patients with high risk of transformation and poor outcome. Leukemia (2012) 1.41
Virus-associated idiopathic thrombocytopenic purpura. Transfus Sci (1998) 1.41
ISBT Code 128 implementation at a regional blood center. Transfusion (2005) 1.41
Human factor IX corrects the bleeding diathesis of mice with hemophilia B. Blood (1998) 1.40
[International diffusion of clinical trials carried out in Spain. An analysis based on their publication in scientific journals]. Med Clin (Barc) (1994) 1.39
[An analysis of Spanish biomedical journals by the impact factor]. Med Clin (Barc) (1992) 1.39
Enforced P-glycoprotein pump function in murine bone marrow cells results in expansion of side population stem cells in vitro and repopulating cells in vivo. Blood (2000) 1.39
A gross and microscopic study of cerebral injuries accompanying maxillofacial high-velocity projectile wounding in dogs. J Oral Maxillofac Surg (1998) 1.39
A comparative study of the skeletal morphology of the temporo-mandibular joint of children and adults. J Postgrad Med (2008) 1.39
Fuzzy control system design via fuzzy Lyapunov functions. IEEE Trans Syst Man Cybern B Cybern (2008) 1.38
An integrated assessment model of carbon sequestration benefits: a case study of Liping county, China. J Environ Manage (2006) 1.38
Hematopoietic stem cell transplantation in chronic lymphocytic leukemia: a report of 12 patients from a single institution. Ann Oncol (1998) 1.37
Five unique compounds: xyloketals from mangrove fungus Xylaria sp. from the South China Sea coast. J Org Chem (2001) 1.37
Evidence from transgenic mice that glucokinase is rate limiting for glucose utilization in the liver. FASEB J (1996) 1.36
Changes in the use of radiotherapy in Ontario 1984-1995. Int J Radiat Oncol Biol Phys (1999) 1.36
Haemophilia B: database of point mutations and short additions and deletions--third edition, 1992. Nucleic Acids Res (1992) 1.36
AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects. Gene Ther (2008) 1.34
PRAD-1/cyclin D1 gene amplification correlates with messenger RNA overexpression and tumor progression in human laryngeal carcinomas. Cancer Res (1994) 1.34
Protection and in vivo selection of hematopoietic stem cells using temozolomide, O6-benzylguanine, and an alkyltransferase-expressing retroviral vector. Mol Ther (2001) 1.33
Series "matrix metalloproteinases in lung health and disease": Matrix metalloproteinases in COPD. Eur Respir J (2011) 1.33