Published in Hum Gene Ther on March 01, 2009
The AAV vector toolkit: poised at the clinical crossroads. Mol Ther (2012) 2.53
Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. Hum Gene Ther (2012) 2.42
Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease. Mol Ther (2011) 1.72
Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice. Hum Gene Ther (2012) 1.55
Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses. Mol Ther (2012) 1.55
Recombinant adeno-associated virus type 2 pseudotypes: comparing safety, specificity, and transduction efficiency in the primate striatum. Laboratory investigation. J Neurosurg (2010) 1.50
AAV9-mediated expression of a non-self protein in nonhuman primate central nervous system triggers widespread neuroinflammation driven by antigen-presenting cell transduction. Mol Ther (2013) 1.39
Functional effects of AAV2-GDNF on the dopaminergic nigrostriatal pathway in parkinsonian rhesus monkeys. Hum Gene Ther (2009) 1.36
Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC. Mol Ther (2010) 1.31
Axonal transport of adeno-associated viral vectors is serotype-dependent. Gene Ther (2012) 1.29
Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates. Mol Ther (2009) 1.14
Biology of adeno-associated viral vectors in the central nervous system. Front Mol Neurosci (2014) 1.04
Optogenetics in the nonhuman primate. Prog Brain Res (2012) 1.04
Adeno-associated virus type 6 is retrogradely transported in the non-human primate brain. Gene Ther (2013) 0.99
Adeno-associated virus serotypes 1, 8, and 9 share conserved mechanisms for anterograde and retrograde axonal transport. Hum Gene Ther (2014) 0.98
Magnetic resonance imaging-guided delivery of adeno-associated virus type 2 to the primate brain for the treatment of lysosomal storage disorders. Hum Gene Ther (2010) 0.97
A next step in adeno-associated virus-mediated gene therapy for neurological diseases: regulation and targeting. Br J Clin Pharmacol (2013) 0.97
Lack of humoral immune response to the tetracycline (Tet) activator in rats injected intracranially with Tet-off rAAV vectors. Gene Ther (2010) 0.92
Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type A. Mol Ther (2012) 0.84
AAV provides an alternative for gene therapy of the peripheral sensory nervous system. Mol Ther (2010) 0.84
Gene therapy for the neurological manifestations in lysosomal storage disorders. J Lipid Res (2014) 0.83
Gene-based therapy of Parkinson's Disease: Translation from animal model to human clinical trial employing convection enhanced delivery. Ann Neurosci (2012) 0.82
Dose-dependent Toxicity of Humanized Renilla reniformis GFP (hrGFP) Limits Its Utility as a Reporter Gene in Mouse Muscle. Mol Ther Nucleic Acids (2013) 0.82
Convection-enhanced delivery of AAV2-PrPshRNA in prion-infected mice. PLoS One (2014) 0.81
Preclinical toxicity evaluation of AAV for pain: evidence from human AAV studies and from the pharmacology of analgesic drugs. Mol Pain (2014) 0.80
Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice. Mol Neurodegener (2016) 0.79
Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease. Mol Ther Methods Clin Dev (2016) 0.77
Robust Long-term Transduction of Common Marmoset Neuromuscular Tissue With rAAV1 and rAAV9. Mol Ther Nucleic Acids (2013) 0.77
Gene therapy for misfolding protein diseases of the central nervous system. Neurotherapeutics (2013) 0.77
High cerebrospinal fluid levels of interleukin-10 attained by AAV in dogs. Gene Ther (2014) 0.76
Strategies for targeting primate neural circuits with viral vectors. J Neurophysiol (2016) 0.76
Antisense gene silencing: therapy for neurodegenerative disorders? Genes (Basel) (2013) 0.75
Gene therapy for metachromatic leukodystrophy. J Neurosci Res (2016) 0.75
Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol (1998) 8.45
Convection-enhanced delivery of macromolecules in the brain. Proc Natl Acad Sci U S A (1994) 6.61
Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells. Proc Natl Acad Sci U S A (1982) 5.73
Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet (1994) 5.49
Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods (2002) 5.30
Rescue of adeno-associated virus from recombinant plasmids: gene correction within the terminal repeats of AAV. Cell (1983) 5.27
Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol Ther (2006) 4.64
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci U S A (2008) 4.53
Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. Proc Natl Acad Sci U S A (2000) 4.39
Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther (2006) 4.36
New recombinant serotypes of AAV vectors. Curr Gene Ther (2005) 3.87
Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach. Exp Neurol (2000) 2.98
Adeno-associated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors. J Virol (2001) 2.95
Alpha2,3 and alpha2,6 N-linked sialic acids facilitate efficient binding and transduction by adeno-associated virus types 1 and 6. J Virol (2006) 2.79
Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC. Mol Ther (2006) 2.41
Immune response to green fluorescent protein: implications for gene therapy. Gene Ther (1999) 2.27
From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy. Curr Gene Ther (2003) 2.12
The "perivascular pump" driven by arterial pulsation is a powerful mechanism for the distribution of therapeutic molecules within the brain. Mol Ther (2006) 2.10
Immunogenicity of enhanced green fluorescent protein (EGFP) in BALB/c mice: identification of an H2-Kd-restricted CTL epitope. Gene Ther (2000) 2.01
Convection-enhanced distribution of large molecules in gray matter during interstitial drug infusion. J Neurosurg (1995) 1.94
Two novel adeno-associated viruses from cynomolgus monkey: pseudotyping characterization of capsid protein. Virology (2004) 1.88
Viral vectors for gene delivery to the nervous system. Nat Rev Neurosci (2003) 1.86
Gadolinium-loaded liposomes allow for real-time magnetic resonance imaging of convection-enhanced delivery in the primate brain. Exp Neurol (2005) 1.78
Real-time visualization and characterization of liposomal delivery into the monkey brain by magnetic resonance imaging. Brain Res Brain Res Protoc (2005) 1.74
Immunity to adeno-associated virus vectors in animals and humans: a continued challenge. Gene Ther (2008) 1.73
Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain. Hum Gene Ther (2006) 1.72
Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography. Hum Gene Ther (2002) 1.60
Targeted retrograde gene delivery for neuronal protection. Mol Ther (2002) 1.59
Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain. Gene Ther (2003) 1.50
Striatal delivery of rAAV-hAADC to rats with preexisting immunity to AAV. Mol Ther (2004) 1.49
AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease. Mol Ther (2005) 1.41
Biodistribution of adeno-associated virus type-2 in nonhuman primates after convection-enhanced delivery to brain. Mol Ther (2008) 1.40
Current status of gene therapy trials for Parkinson's disease. Exp Neurol (2007) 1.34
Convection-enhanced delivery of AAV-2 combined with heparin increases TK gene transfer in the rat brain. Neuroreport (2001) 1.26
Distribution of AAV-TK following intracranial convection-enhanced delivery into rats. Cell Transplant (2000) 1.21
Safety of real-time convection-enhanced delivery of liposomes to primate brain: a long-term retrospective. Exp Neurol (2007) 1.14
Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease. Proc Natl Acad Sci U S A (2005) 1.14
Adeno-associated virus vector-mediated transduction in the cat brain. Gene Ther (2003) 1.14
Oligodendrocyte-specific gene expression in mouse brain: use of a myelin-forming cell type-specific promoter in an adeno-associated virus. J Neurosci Res (1999) 1.11
Astrocytes as antigen-presenting cells: expression of IL-12/IL-23. J Neurochem (2005) 1.05
Recombinant adeno-associated virus serotypes 2- and 5-mediated gene transfer in the mammalian brain: quantitative analysis of heparin co-infusion. Mol Ther (2002) 1.02
Basic fibroblast growth factor enhances transduction, distribution, and axonal transport of adeno-associated virus type 2 vector in rat brain. Hum Gene Ther (2004) 1.00
Role of astrocytes in antigen presentation and naive T-cell activation. J Neuroimmunol (2000) 0.97
Amelioration of arthritis by intraarticular dominant negative Ikk beta gene therapy using adeno-associated virus type 5. Hum Gene Ther (2006) 0.95
Green fluorescent protein expression in dendritic cells enhances their immunogenicity and elicits specific cytotoxic T-cell responses in humans. Exp Hematol (2004) 0.92
RNA polymerase III transcribes human microRNAs. Nat Struct Mol Biol (2006) 6.81
siRNA-mediated gene silencing in vitro and in vivo. Nat Biotechnol (2002) 5.65
Transvascular delivery of small interfering RNA to the central nervous system. Nature (2007) 5.21
RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc Natl Acad Sci U S A (2005) 4.79
RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. Nat Med (2004) 4.63
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci U S A (2008) 4.53
The bifunctional microRNA miR-9/miR-9* regulates REST and CoREST and is downregulated in Huntington's disease. J Neurosci (2008) 3.62
Allele-specific silencing of dominant disease genes. Proc Natl Acad Sci U S A (2003) 3.00
Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo. Mol Ther (2008) 2.66
Structure and activity of putative intronic miRNA promoters. RNA (2010) 2.50
Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. Mol Ther (2009) 2.47
Identification of PDGFR as a receptor for AAV-5 transduction. Nat Med (2003) 2.43
Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. Hum Gene Ther (2012) 2.42
Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC. Mol Ther (2006) 2.41
MicroRNAs potentiate neural development. Neuron (2009) 2.25
The "perivascular pump" driven by arterial pulsation is a powerful mechanism for the distribution of therapeutic molecules within the brain. Mol Ther (2006) 2.10
Reflux-free cannula for convection-enhanced high-speed delivery of therapeutic agents. J Neurosurg (2005) 2.07
Restoring Acid-sensing ion channel-1a in the amygdala of knock-out mice rescues fear memory but not unconditioned fear responses. J Neurosci (2008) 1.90
Platelet-mediated modulation of adaptive immunity. A communication link between innate and adaptive immune compartments. Immunity (2003) 1.84
Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis. Mol Ther (2008) 1.82
Image-guided convection-enhanced delivery platform in the treatment of neurological diseases. Neurotherapeutics (2008) 1.79
Gadolinium-loaded liposomes allow for real-time magnetic resonance imaging of convection-enhanced delivery in the primate brain. Exp Neurol (2005) 1.78
Virus-mediated transduction of murine retina with adeno-associated virus: effects of viral capsid and genome size. J Virol (2002) 1.77
A dose-ranging study of AAV-hAADC therapy in Parkinsonian monkeys. Mol Ther (2006) 1.77
Real-time visualization and characterization of liposomal delivery into the monkey brain by magnetic resonance imaging. Brain Res Brain Res Protoc (2005) 1.74
Targeted viral delivery of Cre recombinase induces conditional gene deletion in cardiovascular circuits of the mouse brain. Physiol Genomics (2004) 1.74
Functional repair of human donor lungs by IL-10 gene therapy. Sci Transl Med (2009) 1.72
Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain. Hum Gene Ther (2006) 1.72
Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease. Mol Ther (2011) 1.72
Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease. Hum Gene Ther (2012) 1.70
Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs. RNA (2008) 1.70
Efficient gene therapy-based method for the delivery of therapeutics to primate cortex. Proc Natl Acad Sci U S A (2009) 1.70
An engineered zinc finger protein activator of the endogenous glial cell line-derived neurotrophic factor gene provides functional neuroprotection in a rat model of Parkinson's disease. J Neurosci (2010) 1.59
Clarifying lysosomal storage diseases. Trends Neurosci (2011) 1.57
CHIP suppresses polyglutamine aggregation and toxicity in vitro and in vivo. J Neurosci (2005) 1.57
Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy. Nat Med (2009) 1.57
Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors. J Neurosci (2005) 1.56
A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration. J Neurosci (2004) 1.56
Gene therapy for lysosomal storage diseases. Mol Ther (2006) 1.55
Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses. Mol Ther (2012) 1.55
Real-time MR imaging of adeno-associated viral vector delivery to the primate brain. Neuroimage (2008) 1.53
Silencing primary dystonia: lentiviral-mediated RNA interference therapy for DYT1 dystonia. J Neurosci (2005) 1.53
Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates. Hum Gene Ther (2013) 1.52
Diverse splicing patterns of exonized Alu elements in human tissues. PLoS Genet (2008) 1.52
Interventional MRI-guided putaminal delivery of AAV2-GDNF for a planned clinical trial in Parkinson's disease. Mol Ther (2011) 1.50
Mutant huntingtin's interaction with mitochondrial protein Drp1 impairs mitochondrial biogenesis and causes defective axonal transport and synaptic degeneration in Huntington's disease. Hum Mol Genet (2011) 1.48
Safety evaluation of AAV2-GDNF gene transfer into the dopaminergic nigrostriatal pathway in aged and parkinsonian rhesus monkeys. Hum Gene Ther (2009) 1.46
Lentivirus vectors pseudotyped with filoviral envelope glycoproteins transduce airway epithelia from the apical surface independently of folate receptor alpha. J Virol (2003) 1.45
Adenosine deamination in human transcripts generates novel microRNA binding sites. Hum Mol Genet (2009) 1.43
Biodistribution of adeno-associated virus type-2 in nonhuman primates after convection-enhanced delivery to brain. Mol Ther (2008) 1.40
Detection of infusate leakage in the brain using real-time imaging of convection-enhanced delivery. J Neurosurg (2008) 1.40
Widespread establishment and regulatory impact of Alu exons in human genes. Proc Natl Acad Sci U S A (2011) 1.40
The gastric HK-ATPase: structure, function, and inhibition. Pflugers Arch (2008) 1.39
Novel nanoliposomal CPT-11 infused by convection-enhanced delivery in intracranial tumors: pharmacology and efficacy. Cancer Res (2006) 1.36
Functional effects of AAV2-GDNF on the dopaminergic nigrostriatal pathway in parkinsonian rhesus monkeys. Hum Gene Ther (2009) 1.36
Current status of gene therapy trials for Parkinson's disease. Exp Neurol (2007) 1.34
Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors. Proc Natl Acad Sci U S A (2002) 1.33
Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis. J Neurosci (2006) 1.33
Striatal volume differences between non-human and human primates. J Neurosci Methods (2008) 1.33
Connecdenn, a novel DENN domain-containing protein of neuronal clathrin-coated vesicles functioning in synaptic vesicle endocytosis. J Neurosci (2006) 1.33
Anterograde axonal transport of AAV2-GDNF in rat basal ganglia. Mol Ther (2010) 1.32
Effects of the perivascular space on convection-enhanced delivery of liposomes in primate putamen. Exp Neurol (2005) 1.32
Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC. Mol Ther (2010) 1.31
Novel platform for MRI-guided convection-enhanced delivery of therapeutics: preclinical validation in nonhuman primate brain. Stereotact Funct Neurosurg (2011) 1.30
Convection-enhanced delivery of Ls-TPT enables an effective, continuous, low-dose chemotherapy against malignant glioma xenograft model. Neuro Oncol (2006) 1.29
Targeting Alzheimer's disease genes with RNA interference: an efficient strategy for silencing mutant alleles. Nucleic Acids Res (2004) 1.27
Selective gene transfer to key cardiovascular regions of the brain: comparison of two viral vector systems. Hypertension (2002) 1.27
Convection-enhanced delivery of a topoisomerase I inhibitor (nanoliposomal topotecan) and a topoisomerase II inhibitor (pegylated liposomal doxorubicin) in intracranial brain tumor xenografts. Neuro Oncol (2006) 1.26
Characterization of a novel potassium-competitive acid blocker of the gastric H,K-ATPase, 1-[5-(2-fluorophenyl)-1-(pyridin-3-ylsulfonyl)-1H-pyrrol-3-yl]-N-methylmethanamine monofumarate (TAK-438). J Pharmacol Exp Ther (2011) 1.26
Convection-enhanced delivery of tumor necrosis factor-related apoptosis-inducing ligand with systemic administration of temozolomide prolongs survival in an intracranial glioblastoma xenograft model. Cancer Res (2004) 1.26
Canine model of convection-enhanced delivery of liposomes containing CPT-11 monitored with real-time magnetic resonance imaging: laboratory investigation. J Neurosurg (2008) 1.25