Published in Hum Gene Ther on April 01, 2009
Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications. Nat Rev Genet (2009) 3.09
In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance. Blood (2009) 1.34
MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression. Mol Ther (2010) 1.30
Strategies to modulate immune responses: a new frontier for gene therapy. Mol Ther (2009) 1.14
Image-guided, intravascular hydrodynamic gene delivery to skeletal muscle in pigs. Mol Ther (2009) 1.08
Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I. Mol Ther (2010) 1.08
Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies. Curr Gene Ther (2011) 1.08
Therapeutics Based on microRNA: A New Approach for Liver Cancer. Curr Genomics (2010) 0.96
Gene therapy with helper-dependent adenoviral vectors: current advances and future perspectives. Viruses (2010) 0.87
Effective Prevention of Liver Fibrosis by Liver-targeted Hydrodynamic Gene Delivery of Matrix Metalloproteinase-13 in a Rat Liver Fibrosis Model. Mol Ther Nucleic Acids (2016) 0.78
Gene therapy for liver regeneration: experimental studies and prospects for clinical trials. World J Gastroenterol (2010) 0.78
Repeatable, Inducible Micro-RNA-Based Technology Tightly Controls Liver Transgene Expression. Mol Ther Nucleic Acids (2014) 0.77
Effects of APC De-targeting and GAr modification on the duration of luciferase expression from plasmid DNA delivered to skeletal muscle. Curr Gene Ther (2015) 0.77
miRNA-mediated silencing in hepatocytes can increase adaptive immune responses to adenovirus vector-delivered transgenic antigens. Mol Ther (2011) 0.76
Effects of transgene expression level per cell in mice livers on induction of transgene-specific immune responses after hydrodynamic gene transfer. Gene Ther (2016) 0.75
Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes. Proc Natl Acad Sci U S A (2007) 3.30
Efficient delivery of siRNA for inhibition of gene expression in postnatal mice. Nat Genet (2002) 3.08
A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs. Mol Ther (2004) 2.16
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Mol Ther (2011) 2.06
Medium chain acyl-coenzyme A dehydrogenase deficiency in a neonate. N Engl J Med (2007) 1.55
Improved nutritional management of phenylketonuria by using a diet containing glycomacropeptide compared with amino acids. Am J Clin Nutr (2009) 1.32
Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study. J Pediatr (2009) 1.27
Mechanism of plasmid delivery by hydrodynamic tail vein injection. I. Hepatocyte uptake of various molecules. J Gene Med (2006) 1.25
Endosomolysis by masking of a membrane-active agent (EMMA) for cytoplasmic release of macromolecules. Bioconjug Chem (2003) 1.18
Delivery of siRNA and siRNA expression constructs to adult mammals by hydrodynamic intravascular injection. Methods Enzymol (2005) 1.10
Transcriptional and phenotypic comparisons of Ppara knockout and siRNA knockdown mice. Nucleic Acids Res (2006) 1.08
L-carnitine administration reduces number of episodes in cyclic vomiting syndrome. Clin Pediatr (Phila) (2002) 1.06
Intraarterial delivery of naked plasmid DNA expressing full-length mouse dystrophin in the mdx mouse model of duchenne muscular dystrophy. Hum Gene Ther (2004) 1.05
Correction of murine PKU following AAV-mediated intramuscular expression of a complete phenylalanine hydroxylating system. Mol Ther (2008) 1.05
Prospective diagnosis of 2-methylbutyryl-CoA dehydrogenase deficiency in the Hmong population by newborn screening using tandem mass spectrometry. Pediatrics (2003) 1.05
The effect of cell division on the cellular dynamics of microinjected DNA and dextran. Mol Ther (2002) 1.04
Mechanism of plasmid delivery by hydrodynamic tail vein injection. II. Morphological studies. J Gene Med (2006) 1.04
Systemic siRNA delivery via hydrodynamic intravascular injection. Adv Drug Deliv Rev (2007) 1.03
Condensation of nonstochiometric DNA/polycation complexes by divalent cations. Biopolymers (2006) 1.00
Evaluation of hydrodynamic limb vein injections in nonhuman primates. Hum Gene Ther (2010) 0.98
Use of Evans blue dye to compare limb muscles in exercised young and old mdx mice. Muscle Nerve (2010) 0.94
Sustained liver-specific transgene expression from the albumin promoter in mice following hydrodynamic plasmid DNA delivery. J Gene Med (2008) 0.94
The role of molecular testing and enzyme analysis in the management of hypomorphic citrullinemia. Am J Med Genet A (2008) 0.92
Membrane activity and transfection ability of amphipathic polycations as a function of alkyl group size. Bioconjug Chem (2005) 0.90
Surgical procedures for intravascular delivery of plasmid DNA to organs. Methods Enzymol (2002) 0.89
Genetic immunization for antibody generation in research animals by intravenous delivery of plasmid DNA. Biotechniques (2006) 0.88
Progress toward a nonviral gene therapy protocol for the treatment of anemia. Hum Gene Ther (2007) 0.88
Magnetic resonance imaging-monitored plasmid DNA delivery in primate limb muscle. Hum Gene Ther (2007) 0.88
2-methylbutyryl-CoA dehydrogenase deficiency in Hmong infants identified by expanded newborn screen. WMJ (2007) 0.85
Functional efficacy of dystrophin expression from plasmids delivered to mdx mice by hydrodynamic limb vein injection. Hum Gene Ther (2010) 0.85
Dose response in rodents and nonhuman primates after hydrodynamic limb vein delivery of naked plasmid DNA. Hum Gene Ther (2011) 0.83
In vivo selection and validation of liver-specific ligands using a new T7 phage peptide display system. Drug Deliv (2007) 0.83
A single acyl-CoA dehydrogenase is required for catabolism of isoleucine, valine and short-chain fatty acids in Aspergillus nidulans. Fungal Genet Biol (2007) 0.83
Expression of phenylalanine hydroxylase (PAH) in erythrogenic bone marrow does not correct hyperphenylalaninemia in Pah(enu2) mice. J Gene Med (2003) 0.82
Entrapment and condensation of DNA in neutral reverse micelles. Biophys J (2002) 0.80
Naked DNA gene transfer in mammalian cells. Methods Mol Biol (2004) 0.79
Muscle damage after delivery of naked plasmid DNA into skeletal muscles is batch dependent. Hum Gene Ther (2011) 0.79
Hybrid PET/CT for noninvasive pharmacokinetic evaluation of dynamic PolyConjugates, a synthetic siRNA delivery system. Bioconjug Chem (2010) 0.79
A new peptide ligand that targets particles and heterologous proteins to hepatocytes in vivo. Mol Ther (2003) 0.79
Hepatocyte targeting of nucleic acid complexes and liposomes by a T7 phage p17 peptide. Mol Pharm (2006) 0.78
Systemic gene transfer to skeletal muscle using reengineered AAV vectors. Methods Mol Biol (2011) 0.77
Efficient in vitro and in vivo expression of covalently modified plasmid DNA. Mol Ther (2003) 0.77
The role of a microscopic colloidally stabilized phase in solubilizing oligoamine-condensed DNA complexes. Biophys J (2003) 0.75
Novel human pathological mutations. Gene symbol: ASS1. Disease: Citrullinaemia. Hum Genet (2009) 0.75
Peptide-mediated targeting of hepatocytes via low density lipoprotein receptor-related protein (LRP). Drug Deliv (2009) 0.75
Novel human pathological mutations. Gene symbol: ASS1. Disease: Citrullinaemia. Hum Genet (2009) 0.75
Rapidly reversible hydrophobization: an approach to high first-pass drug extraction. Chem Biol (2007) 0.75