Published in Hum Mol Genet on October 01, 2002
Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart. Circulation (2003) 2.22
Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury. Mol Ther (2005) 2.08
DNA-based therapeutics and DNA delivery systems: a comprehensive review. AAPS J (2005) 2.03
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
Dystrophin deficiency in Drosophila reduces lifespan and causes a dilated cardiomyopathy phenotype. Aging Cell (2008) 1.52
Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy. Dis Model Mech (2015) 1.30
Emerging strategies for cell and gene therapy of the muscular dystrophies. Expert Rev Mol Med (2009) 1.29
Viral-mediated gene therapy for the muscular dystrophies: successes, limitations and recent advances. Biochim Biophys Acta (2006) 1.25
Full-length dystrophin expression in half of the heart cells ameliorates beta-isoproterenol-induced cardiomyopathy in mdx mice. Hum Mol Genet (2004) 1.25
Trans-splicing adeno-associated viral vector-mediated gene therapy is limited by the accumulation of spliced mRNA but not by dual vector coinfection efficiency. Hum Gene Ther (2004) 1.16
Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy. Mol Ther (2013) 1.13
Interleukin-15 administration improves diaphragm muscle pathology and function in dystrophic mdx mice. Am J Pathol (2005) 1.10
Inhibitory control over Ca(2+) sparks via mechanosensitive channels is disrupted in dystrophin deficient muscle but restored by mini-dystrophin expression. PLoS One (2008) 1.08
Gene therapy in large animal models of muscular dystrophy. ILAR J (2009) 1.05
Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy. Hum Mol Genet (2013) 0.99
Truncated dystrophins can influence neuromuscular synapse structure. Mol Cell Neurosci (2009) 0.97
Progress in gene therapy of dystrophic heart disease. Gene Ther (2012) 0.96
Synthetic intron improves transduction efficiency of trans-splicing adeno-associated viral vectors. Hum Gene Ther (2006) 0.95
L-type Ca2+ channel function is linked to dystrophin expression in mammalian muscle. PLoS One (2008) 0.92
Proteomic profiling of antisense-induced exon skipping reveals reversal of pathobiochemical abnormalities in dystrophic mdx diaphragm. Proteomics (2009) 0.91
Restoration of gamma-sarcoglycan localization and mechanical signal transduction are independent in murine skeletal muscle. J Biol Chem (2010) 0.87
Duchenne muscular dystrophy gene therapy in the canine model. Hum Gene Ther Clin Dev (2015) 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
Incorporation of DOPE into Lipoplexes formed from a Ferrocenyl Lipid leads to Inverse Hexagonal Nanostructures that allow Redox-Based Control of Transfection in High Serum. Soft Matter (2012) 0.83
Gene therapy to treat cardiac arrhythmias. Nat Rev Cardiol (2015) 0.83
Exploration of lipid metabolism in relation with plasma membrane properties of Duchenne muscular dystrophy cells: influence of L-carnitine. PLoS One (2012) 0.83
Differential myofiber-type transduction preference of adeno-associated virus serotypes 6 and 9. Skelet Muscle (2015) 0.80
Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials. Am J Transl Res (2016) 0.80
Dystrophin Gene Replacement and Gene Repair Therapy for Duchenne Muscular Dystrophy in 2016: An Interview. Hum Gene Ther Clin Dev (2016) 0.79
Thermodynamic stability, unfolding kinetics, and aggregation of the N-terminal actin-binding domains of utrophin and dystrophin. Proteins (2012) 0.79
Systemic delivery of adeno-associated viral vectors. Curr Opin Virol (2016) 0.78
The FVB Background Does Not Dramatically Alter the Dystrophic Phenotype of Mdx Mice. PLoS Curr (2015) 0.77
Gene therapy in monogenic congenital myopathies. Methods (2015) 0.76
Genome Editing Gene Therapy for Duchenne Muscular Dystrophy. J Neuromuscul Dis (2015) 0.75
Impact of intravenous infusion time on AAV8 vector pharmacokinetics, safety, and liver transduction in cynomolgus macaques. Mol Ther Methods Clin Dev (2016) 0.75
DNA as therapeutics; an update. Indian J Pharm Sci (2009) 0.75