Published in Curr Opin Virol on July 25, 2016
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 | NCT02122952
In vivo myomaker-mediated heterologous fusion and nuclear reprogramming. FASEB J (2016) 0.75
Nanotherapy for Duchenne muscular dystrophy. Wiley Interdiscip Rev Nanomed Nanobiotechnol (2017) 0.75
A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy. Mol Ther Methods Clin Dev (2017) 0.75
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A (2002) 11.11
ADENOVIRUS-ASSOCIATED DEFECTIVE VIRUS PARTICLES. Science (1965) 9.77
Clades of Adeno-associated viruses are widely disseminated in human tissues. J Virol (2004) 7.12
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol (2008) 6.82
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol (1996) 6.43
Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr Top Microbiol Immunol (1992) 6.16
Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol Ther (2008) 5.83
Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med (2004) 5.59
Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat Biotechnol (2005) 5.27
Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci U S A (1996) 4.76
Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2. J Virol (1998) 4.71
Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol Ther (2006) 4.64
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol (2010) 4.62
Widespread long-term gene transfer to mouse skeletal muscles and heart. J Clin Invest (1992) 4.43
Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther (2006) 4.36
Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis (2009) 4.27
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science (2015) 4.18
Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue. J Virol (1998) 4.17
Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat Biotechnol (2006) 4.12
New recombinant serotypes of AAV vectors. Curr Gene Ther (2005) 3.87
Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci U S A (2008) 3.84
In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J Virol (2008) 3.79
Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ Res (2006) 3.63
Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors. Hum Gene Ther (2010) 3.37
Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum Gene Ther (1996) 3.26
rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med (2006) 3.13
Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Mol Ther (2011) 2.91
Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med (2006) 2.78
Effect of genome size on AAV vector packaging. Mol Ther (2009) 2.77
Transcytosis: crossing cellular barriers. Physiol Rev (2003) 2.62
Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat. Hum Gene Ther (2008) 2.27
Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice. Hum Mol Genet (2010) 2.26
A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs. Mol Ther (2008) 2.22
Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors. Nat Biotechnol (2005) 2.16
Engineering adeno-associated viruses for clinical gene therapy. Nat Rev Genet (2014) 2.16
Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector. Nat Med (1999) 2.07
Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle. Nat Biotechnol (2009) 2.07
Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization. J Virol (2006) 2.07
Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy. Sci Transl Med (2010) 2.02
Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration. Gene Ther (2007) 2.01
Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets. Hum Gene Ther (2007) 1.95
Intracellular trafficking of adeno-associated viral vectors. Gene Ther (2005) 1.95
Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery. Hum Mol Genet (2010) 1.89
Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer. Mol Ther (2011) 1.86
A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection. Proc Natl Acad Sci U S A (2009) 1.85
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature (2013) 1.80
Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery. Mol Ther (2011) 1.79
Characterization of adeno-associated virus genomes isolated from human tissues. J Virol (2005) 1.72
Characterization of genome integrity for oversized recombinant AAV vector. Mol Ther (2009) 1.71
An essential receptor for adeno-associated virus infection. Nature (2016) 1.69
Prevention of cardiomyopathy in delta-sarcoglycan knockout mice after systemic transfer of targeted adeno-associated viral vectors. Cardiovasc Res (2009) 1.67
Myostatin propeptide gene delivery by adeno-associated virus serotype 8 vectors enhances muscle growth and ameliorates dystrophic phenotypes in mdx mice. Hum Gene Ther (2008) 1.65
Prevention of dystrophin-deficient cardiomyopathy in twenty-one-month-old carrier mice by mosaic dystrophin expression or complementary dystrophin/utrophin expression. Circ Res (2007) 1.65
Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb. Mol Ther (2009) 1.64
Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle. Mol Ther (2008) 1.63
AAV transcytosis through barrier epithelia and endothelium. Mol Ther (2005) 1.60
Robust cardiomyocyte-specific gene expression following systemic injection of AAV: in vivo gene delivery follows a Poisson distribution. Gene Ther (2010) 1.58
Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs. Mol Ther (2010) 1.56
Systemic Trans-splicing adeno-associated viral delivery efficiently transduces the heart of adult mdx mouse, a model for duchenne muscular dystrophy. Hum Gene Ther (2009) 1.53
Efficient whole-body transduction with trans-splicing adeno-associated viral vectors. Mol Ther (2007) 1.48
Intracellular transport of recombinant adeno-associated virus vectors. Gene Ther (2012) 1.44
Challenges and opportunities in dystrophin-deficient cardiomyopathy gene therapy. Hum Mol Genet (2006) 1.44
Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle. J Virol (2008) 1.44
Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer. Hum Mol Genet (2010) 1.43
Tailoring the AAV vector capsid for gene therapy. Gene Ther (2008) 1.41
Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10. Mol Ther (2014) 1.41
Lessons learned from the gene therapy trial for ornithine transcarbamylase deficiency. Mol Genet Metab (2009) 1.41
Gene therapy of muscular dystrophy. Hum Mol Genet (2002) 1.38
AAV capsid structure and cell interactions. Methods Mol Biol (2011) 1.36
Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery. Mol Ther (2009) 1.35
Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy. Sci Transl Med (2014) 1.32
Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors. Mol Ther (2007) 1.31
Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vector. Circulation (2005) 1.30
Adeno-associated virus and the development of adeno-associated virus vectors: a historical perspective. Mol Ther (2004) 1.30
Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice. Mol Ther (2008) 1.29
The role of the adeno-associated virus capsid in gene transfer. Methods Mol Biol (2008) 1.29
A 10 patient case report on the impact of plasmapheresis upon neutralizing factors against adeno-associated virus (AAV) types 1, 2, 6, and 8. Mol Ther (2011) 1.29
Human hepatocyte growth factor receptor is a cellular coreceptor for adeno-associated virus serotype 3. Hum Gene Ther (2010) 1.26
Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle. Hum Gene Ther (2010) 1.26
Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle. Mol Ther (2009) 1.21
Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle. Mol Ther (2008) 1.21
Hydrodynamic limb vein injection of adeno-associated virus serotype 8 vector carrying canine myostatin propeptide gene into normal dogs enhances muscle growth. Hum Gene Ther (2009) 1.21
Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6. Mol Ther (2010) 1.21
Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping. Mol Ther (2012) 1.18
Intravenous gene therapy with PIM-1 via a cardiotropic viral vector halts the progression of diabetic cardiomyopathy through promotion of prosurvival signaling. Circ Res (2011) 1.18
Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients. Mol Ther (2014) 1.17
Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus. Hum Mol Genet (2015) 1.16
Comparative analysis of adeno-associated virus capsid stability and dynamics. J Virol (2013) 1.16
AAV-Mediated Gene Therapy for Research and Therapeutic Purposes. Annu Rev Virol (2014) 1.13
Phenotypic correction of alpha-sarcoglycan deficiency by intra-arterial injection of a muscle-specific serotype 1 rAAV vector. Mol Ther (2007) 1.13
Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B. Mol Ther (2012) 1.13
Distinct immune responses to transgene products from rAAV1 and rAAV8 vectors. Proc Natl Acad Sci U S A (2009) 1.11
AAV-microdystrophin therapy improves cardiac performance in aged female mdx mice. Mol Ther (2011) 1.08
Enhancing the Clinical Potential of AAV Vectors by Capsid Engineering to Evade Pre-Existing Immunity. Front Microbiol (2011) 1.08
Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue. Gene Ther (2012) 1.06
Expanding adeno-associated viral vector capacity: a tale of two vectors. Biotechnol Genet Eng Rev (2007) 1.06
Modulation of Starling forces and muscle fiber maturity permits adenovirus-mediated gene transfer to adult dystrophic (mdx) mice by the intravascular route. Hum Gene Ther (2000) 1.06
Glycan binding avidity determines the systemic fate of adeno-associated virus type 9. J Virol (2012) 1.05
Effective delivery of large genes to the retina by dual AAV vectors. EMBO Mol Med (2013) 1.04
Drawing a high-resolution functional map of adeno-associated virus capsid by massively parallel sequencing. Nat Commun (2014) 1.04