Published in Mol Ther on January 01, 2003
Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Hum Gene Ther (2010) 2.51
Assessing the potential for AAV vector genotoxicity in a murine model. Blood (2010) 2.48
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
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. Blood (2007) 2.05
Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods. Hum Gene Ther Methods (2012) 1.79
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Hum Gene Ther (2010) 1.51
Intraganglionic AAV6 results in efficient and long-term gene transfer to peripheral sensory nervous system in adult rats. PLoS One (2013) 1.51
Heparin binding induces conformational changes in Adeno-associated virus serotype 2. J Struct Biol (2008) 1.44
AAV2-mediated gene delivery to monkey putamen: evaluation of an infusion device and delivery parameters. Exp Neurol (2005) 1.29
Viral-mediated gene therapy for the muscular dystrophies: successes, limitations and recent advances. Biochim Biophys Acta (2006) 1.25
Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses. Mol Ther (2008) 1.11
The gene therapy journey for hemophilia: are we there yet? Blood (2012) 1.10
Absolute determination of single-stranded and self-complementary adeno-associated viral vector genome titers by droplet digital PCR. Hum Gene Ther Methods (2014) 1.08
Organ-specific shifts in mtDNA heteroplasmy following systemic delivery of a mitochondria-targeted restriction endonuclease. Gene Ther (2010) 1.06
Transient transfection methods for clinical adeno-associated viral vector production. Hum Gene Ther (2009) 1.05
Predictors of hepatitis B cure using gene therapy to deliver DNA cleavage enzymes: a mathematical modeling approach. PLoS Comput Biol (2013) 1.05
Novel caprine adeno-associated virus (AAV) capsid (AAV-Go.1) is closely related to the primate AAV-5 and has unique tropism and neutralization properties. J Virol (2005) 1.04
Limitations of encapsidation of recombinant self-complementary adeno-associated viral genomes in different serotype capsids and their quantitation. Hum Gene Ther Methods (2012) 1.04
Proteolytic mapping of the adeno-associated virus capsid. Mol Ther (2006) 1.03
Host and vector-dependent effects on the risk of germline transmission of AAV vectors. Mol Ther (2009) 1.00
Analysis of particle content of recombinant adeno-associated virus serotype 8 vectors by ion-exchange chromatography. Hum Gene Ther Methods (2012) 0.94
Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material. Hum Gene Ther (2014) 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
Near-perfect infectivity of wild-type AAV as benchmark for infectivity of recombinant AAV vectors. Gene Ther (2010) 0.91
Enhanced real-time monitoring of adeno-associated virus trafficking by virus-quantum dot conjugates. ACS Nano (2011) 0.90
Safety of liver gene transfer following peripheral intravascular delivery of adeno-associated virus (AAV)-5 and AAV-6 in a large animal model. Hum Gene Ther (2011) 0.89
AAV-mediated expression of an ADAMTS13 variant prevents shigatoxin-induced thrombotic thrombocytopenic purpura. Blood (2013) 0.87
Viral vectors for in vivo gene transfer in Parkinson's disease: properties and clinical grade production. Exp Neurol (2007) 0.86
Systematic Comparison and Validation of Quantitative Real-Time PCR Methods for the Quantitation of Adeno-Associated Viral Products. Hum Gene Ther Methods (2015) 0.86
Abrogation of β-catenin signaling in oligodendrocyte precursor cells reduces glial scarring and promotes axon regeneration after CNS injury. J Neurosci (2014) 0.85
Enhancing gene delivery of adeno-associated viruses by cell-permeable peptides. Mol Ther Methods Clin Dev (2014) 0.83
Preclinical evaluation of an anti-HCV miRNA cluster for treatment of HCV infection. Mol Ther (2013) 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
A pseudo-plaque method for infectious particle assay and clonal isolation of adeno-associated virus. J Virol Methods (2010) 0.78
Highly divergent integration profile of adeno-associated virus serotype 5 revealed by high-throughput sequencing. J Virol (2013) 0.77
A reliable and feasible qPCR strategy for titrating AAV vectors. Med Sci Monit Basic Res (2013) 0.77
Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med (2008) 17.21
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med (2006) 13.51
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet (2009) 6.71
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood (2002) 4.60
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther (2009) 3.93
AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med (2012) 2.87
Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood (2003) 2.74
Assessing the potential for AAV vector genotoxicity in a murine model. Blood (2010) 2.48
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. Blood (2007) 2.05
Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2. Ophthalmology (2013) 1.85
Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods. Hum Gene Ther Methods (2012) 1.79
A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction. J Virol (2002) 1.71
Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses. Mol Ther (2012) 1.55
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Hum Gene Ther (2010) 1.51
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B. Blood (2010) 1.50
Striatal delivery of rAAV-hAADC to rats with preexisting immunity to AAV. Mol Ther (2004) 1.49
The glial modulatory drug AV411 attenuates mechanical allodynia in rat models of neuropathic pain. Neuron Glia Biol (2006) 1.43
Biodistribution of adeno-associated virus type-2 in nonhuman primates after convection-enhanced delivery to brain. Mol Ther (2008) 1.40
Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs. Mol Ther (2010) 1.35
AAV2-mediated gene delivery to monkey putamen: evaluation of an infusion device and delivery parameters. Exp Neurol (2005) 1.29
Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector. Blood (2003) 1.25
Identification of factors that contribute to recombinant AAV2 particle aggregation and methods to prevent its occurrence during vector purification and formulation. Mol Ther (2005) 1.22
Translational nanomedicine: status assessment and opportunities. Nanomedicine (2009) 1.17
Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography. J Virol Methods (2006) 1.15
Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII. Blood (2003) 1.15
Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B. Mol Ther (2012) 1.13
Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses. Mol Ther (2008) 1.11
Infectious titer assay for adeno-associated virus vectors with sensitivity sufficient to detect single infectious events. Hum Gene Ther (2004) 1.10
Discovery of natural perchlorate in the Antarctic Dry Valleys and its global implications. Environ Sci Technol (2010) 1.02
Host and vector-dependent effects on the risk of germline transmission of AAV vectors. Mol Ther (2009) 1.00
Adeno-associated virus (AAV) capsid genes isolated from rat and mouse liver genomic DNA define two new AAV species distantly related to AAV-5. Virology (2006) 0.92
Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material. Hum Gene Ther (2014) 0.91
Learning to live on a Mars day: fatigue countermeasures during the Phoenix Mars Lander mission. Sleep (2012) 0.90
Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model. Mol Ther (2012) 0.89
Safety of liver gene transfer following peripheral intravascular delivery of adeno-associated virus (AAV)-5 and AAV-6 in a large animal model. Hum Gene Ther (2011) 0.89
The Icebreaker Life Mission to Mars: a search for biomolecular evidence for life. Astrobiology (2013) 0.89
Efficient AAV1-AAV2 hybrid vector for gene therapy of hemophilia. Hum Gene Ther (2006) 0.88
AAV-mediated expression of an ADAMTS13 variant prevents shigatoxin-induced thrombotic thrombocytopenic purpura. Blood (2013) 0.87
Specific adeno-associated virus serotypes facilitate efficient gene transfer into human and non-human primate mesenchymal stromal cells. J Gene Med (2007) 0.85
PEG-modulated column chromatography for purification of recombinant adeno-associated virus serotype 9. J Virol Methods (2011) 0.84
In vitro characterization of the activity of PF-05095808, a novel biological agent for hepatitis C virus therapy. Antimicrob Agents Chemother (2011) 0.84
Adeno-associated viral vector manufacturing: keeping pace with accelerating clinical development. Hum Gene Ther (2011) 0.81
Advancing translational research through the NHLBI Gene Therapy Resource Program (GTRP). Hum Gene Ther Clin Dev (2013) 0.80