Published in Methods Mol Biol on January 01, 2012
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Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol Ther (2011) 3.10
Multiple vitamin K-dependent coagulation zymogens promote adenovirus-mediated gene delivery to hepatocytes. Blood (2006) 2.67
Identification of coagulation factor (F)X binding sites on the adenovirus serotype 5 hexon: effect of mutagenesis on FX interactions and gene transfer. Blood (2009) 2.55
The CD46-Jagged1 interaction is critical for human TH1 immunity. Nat Immunol (2012) 1.97
Stable gene transfer to muscle using non-integrating lentiviral vectors. Mol Ther (2007) 1.81
Codon optimization of human factor VIII cDNAs leads to high-level expression. Blood (2010) 1.68
Increased secretion of lipoproteins in transgenic mice expressing human D374Y PCSK9 under physiological genetic control. Arterioscler Thromb Vasc Biol (2010) 1.68
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Mitochondria and quality control defects in a mouse model of Gaucher disease--links to Parkinson's disease. Cell Metab (2013) 1.32
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The influence of blood on in vivo adenovirus bio-distribution and transduction. Mol Ther (2007) 1.16
Influence of coagulation factor zymogens on the infectivity of adenoviruses pseudotyped with fibers from subgroup D. J Virol (2007) 1.09
Activation and deactivation of periventricular white matter phagocytes during postnatal mouse development. Glia (2010) 1.06
The cyclopentenone 15-deoxy-delta 12,14-prostaglandin J(2) delays lipopolysaccharide-induced preterm delivery and reduces mortality in the newborn mouse. Endocrinology (2008) 1.03
Effect of neutralizing sera on factor x-mediated adenovirus serotype 5 gene transfer. J Virol (2008) 1.02
Activator protein 1 is a key terminal mediator of inflammation-induced preterm labor in mice. FASEB J (2014) 1.02
Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques. Mol Ther (2011) 1.00
Differentiation of human fetal mesenchymal stem cells into cells with an oligodendrocyte phenotype. Cell Cycle (2009) 0.98
β-Glucosidase 2 (GBA2) activity and imino sugar pharmacology. J Biol Chem (2013) 0.97
Influence of coagulation factor x on in vitro and in vivo gene delivery by adenovirus (Ad) 5, Ad35, and chimeric Ad5/Ad35 vectors. Mol Ther (2009) 0.95
Luciferin detection after intranasal vector delivery is improved by intranasal rather than intraperitoneal luciferin administration. Hum Gene Ther (2008) 0.94
Current therapies for the soluble lysosomal forms of neuronal ceroid lipofuscinosis. Biochem Soc Trans (2010) 0.93
Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver. J Mol Med (Berl) (2011) 0.91
LDLR-Gene therapy for familial hypercholesterolaemia: problems, progress, and perspectives. Int Arch Med (2010) 0.91
Delivery and long-term expression of a 135 kb LDLR genomic DNA locus in vivo by hydrodynamic tail vein injection. J Gene Med (2007) 0.91
AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage. Blood (2011) 0.89
Desmin-regulated lentiviral vectors for skeletal muscle gene transfer. Mol Ther (2009) 0.87
Increased glucocerebrosidase (GBA) 2 activity in GBA1 deficient mice brains and in Gaucher leucocytes. J Inherit Metab Dis (2012) 0.86
Exon skipping of hepatic APOB pre-mRNA with splice-switching oligonucleotides reduces LDL cholesterol in vivo. Mol Ther (2013) 0.86
Neonatal gene therapy of glycogen storage disease type Ia using a feline immunodeficiency virus-based vector. Mol Ther (2010) 0.84
Perinatal gene transfer to the liver. Curr Pharm Des (2011) 0.84
The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis. Mol Ther (2012) 0.82
The case for intrauterine gene therapy. Best Pract Res Clin Obstet Gynaecol (2012) 0.81
Fetal gene therapy: recent advances and current challenges. Expert Opin Biol Ther (2011) 0.80
The concept of prenatal gene therapy. Methods Mol Biol (2012) 0.80
In utero gene transfer to the mouse nervous system. Biochem Soc Trans (2010) 0.79
Animal models for prenatal gene therapy: rodent models for prenatal gene therapy. Methods Mol Biol (2012) 0.79
Monitoring for potential adverse effects of prenatal gene therapy: use of large animal models with relevance to human application. Methods Mol Biol (2012) 0.78
The differentiation and engraftment potential of mouse hematopoietic stem cells is maintained after bio-electrospray. Analyst (2009) 0.77
Perinatal gene delivery to the CNS. Ther Deliv (2011) 0.76
Choice of surrogate and physiological markers for prenatal gene therapy. Methods Mol Biol (2012) 0.75
Targeting the respiratory muscles of fetal sheep for prenatal gene therapy for Duchenne muscular dystrophy. Am J Obstet Gynecol (2005) 0.75
Monitoring for potential adverse effects of prenatal gene therapy: mouse models for developmental aberrations and inadvertent germ line transmission. Methods Mol Biol (2012) 0.75
Erratum: Lentiviral vectors can be used for full-length dystrophin gene therapy. Sci Rep (2017) 0.75
Vector systems for prenatal gene therapy: choosing vectors for different applications. Methods Mol Biol (2012) 0.75