Published in Nat Biotechnol on January 22, 2006
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol (2008) 6.82
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol (2010) 4.62
In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J Virol (2008) 3.79
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The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9. J Virol (2006) 2.94
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Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles. Mol Ther (2008) 2.19
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Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina. Mol Ther (2010) 1.77
DNA shuffling of adeno-associated virus yields functionally diverse viral progeny. Mol Ther (2008) 1.70
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Molecular evolution of adeno-associated virus for enhanced glial gene delivery. Mol Ther (2009) 1.57
Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy. Nat Med (2009) 1.57
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Directed evolution of adeno-associated virus to an infectious respiratory virus. Proc Natl Acad Sci U S A (2009) 1.42
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Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain. Nat Biotechnol (2016) 1.11
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Advances in AAV vector development for gene therapy in the retina. Adv Exp Med Biol (2014) 0.94
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AAV ancestral reconstruction library enables selection of broadly infectious viral variants. Gene Ther (2015) 0.91
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An emerging adeno-associated viral vector pipeline for cardiac gene therapy. Hum Gene Ther (2013) 0.88
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Directed evolution of adeno-associated virus for glioma cell transduction. J Neurooncol (2009) 0.86
A versatile adeno-associated virus vector producer cell line method for scalable vector production of different serotypes. Hum Gene Ther (2011) 0.86
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Enhanced sialic acid-dependent endocytosis explains the increased efficiency of infection of airway epithelia by a novel adeno-associated virus. J Virol (2011) 0.84
Essential role of presynaptic NMDA receptors in activity-dependent BDNF secretion and corticostriatal LTP. Neuron (2014) 0.84
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A brain microvasculature endothelial cell-specific viral vector with the potential to treat neurovascular and neurological diseases. EMBO Mol Med (2016) 0.82
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Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors. Curr Gene Ther (2012) 0.81
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The structure of AAVrh32.33, a novel gene delivery vector. J Struct Biol (2014) 0.81
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Improving clinical efficacy of adeno associated vectors by rational capsid bioengineering. J Biomed Sci (2014) 0.80
Pulmonary Targeting of Adeno-associated Viral Vectors by Next-generation Sequencing-guided Screening of Random Capsid Displayed Peptide Libraries. Mol Ther (2016) 0.79
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Engineering and evolution of synthetic adeno-associated virus (AAV) gene therapy vectors via DNA family shuffling. J Vis Exp (2012) 0.79
Characterization of the Adeno-Associated Virus 1 and 6 Sialic Acid Binding Site. J Virol (2016) 0.79
Gene delivery strategies to promote spinal cord repair. Biomark Insights (2015) 0.79
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol (2008) 6.82
Stochastic gene expression in a lentiviral positive-feedback loop: HIV-1 Tat fluctuations drive phenotypic diversity. Cell (2005) 5.71
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol (2010) 4.62
Sonic hedgehog regulates adult neural progenitor proliferation in vitro and in vivo. Nat Neurosci (2003) 3.98
Substrate modulus directs neural stem cell behavior. Biophys J (2008) 3.81
Astrocytes from familial and sporadic ALS patients are toxic to motor neurons. Nat Biotechnol (2011) 3.45
A role for glia in the progression of Rett's syndrome. Nature (2011) 3.18
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
Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors. Proc Natl Acad Sci U S A (2008) 2.31
Optogenetic protein clustering and signaling activation in mammalian cells. Nat Methods (2013) 2.22
Follistatin gene delivery enhances muscle growth and strength in nonhuman primates. Sci Transl Med (2009) 2.21
Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders. Mol Ther (2011) 2.20
IGF-I instructs multipotent adult neural progenitor cells to become oligodendrocytes. J Cell Biol (2004) 2.02
Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis. Proc Natl Acad Sci U S A (2006) 1.97
PI3K/Akt and CREB regulate adult neural hippocampal progenitor proliferation and differentiation. Dev Neurobiol (2007) 1.93
Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery. Hum Mol Genet (2010) 1.89
In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous. Sci Transl Med (2013) 1.86
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The adult substantia nigra contains progenitor cells with neurogenic potential. J Neurosci (2002) 1.78
Transforming growth factor-beta and notch signaling mediate stem cell differentiation into smooth muscle cells. Stem Cells (2010) 1.74
Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous. Mol Ther (2009) 1.73
Designing synthetic materials to control stem cell phenotype. Curr Opin Chem Biol (2007) 1.73
Relative roles of TGF-beta1 and Wnt in the systemic regulation and aging of satellite cell responses. Aging Cell (2009) 1.72
Nox2 redox signaling maintains essential cell populations in the brain. Nat Chem Biol (2010) 1.70
DNA shuffling of adeno-associated virus yields functionally diverse viral progeny. Mol Ther (2008) 1.70
The influence of hydrogel modulus on the proliferation and differentiation of encapsulated neural stem cells. Biomaterials (2009) 1.63
The sonic hedgehog signaling system as a bistable genetic switch. Biophys J (2004) 1.61
Kinetic analysis and modeling of firefly luciferase as a quantitative reporter gene in live mammalian cells. Biotechnol Bioeng (2004) 1.59
Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity. Mol Ther (2008) 1.57
Molecular evolution of adeno-associated virus for enhanced glial gene delivery. Mol Ther (2009) 1.57
Biophysical regulation of epigenetic state and cell reprogramming. Nat Mater (2013) 1.55
Control of stochastic gene expression by host factors at the HIV promoter. PLoS Pathog (2009) 1.50
Designer gene delivery vectors: molecular engineering and evolution of adeno-associated viral vectors for enhanced gene transfer. Pharm Res (2007) 1.50
A degradable polyethylenimine derivative with low toxicity for highly efficient gene delivery. Bioconjug Chem (2003) 1.50
Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis. Ann Neurol (2005) 1.44
Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS. Proc Natl Acad Sci U S A (2013) 1.44
Signal dynamics in Sonic hedgehog tissue patterning. Development (2006) 1.43
Characterization of integrin engagement during defined human embryonic stem cell culture. FASEB J (2009) 1.43
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Combinatorial latency reactivation for HIV-1 subtypes and variants. J Virol (2010) 1.40
A novel adeno-associated viral variant for efficient and selective intravitreal transduction of rat Müller cells. PLoS One (2009) 1.40
A global assessment of stem cell engineering. Tissue Eng Part A (2014) 1.38
A conditional deletion of the NR1 subunit of the NMDA receptor in adult spinal cord dorsal horn reduces NMDA currents and injury-induced pain. J Neurosci (2003) 1.38
A fully defined and scalable 3D culture system for human pluripotent stem cell expansion and differentiation. Proc Natl Acad Sci U S A (2013) 1.38
Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS. Mol Ther (2013) 1.36
Theoretical design of a gene therapy to prevent AIDS but not human immunodeficiency virus type 1 infection. J Virol (2003) 1.34
Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease. Muscle Nerve (2009) 1.34
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Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome. J Neurosci (2013) 1.26
Development of a low bias method for characterizing viral populations using next generation sequencing technology. PLoS One (2010) 1.25
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AAV4-mediated expression of IGF-1 and VEGF within cellular components of the ventricular system improves survival outcome in familial ALS mice. Mol Ther (2010) 1.22
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mdx(⁵cv) mice manifest more severe muscle dysfunction and diaphragm force deficits than do mdx Mice. Am J Pathol (2011) 0.96
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