Published in J Gene Med on July 01, 2010
Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery. Mol Ther (2011) 1.79
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy. J Clin Invest (2013) 1.44
Female mucopolysaccharidosis IIIA mice exhibit hyperactivity and a reduced sense of danger in the open field test. PLoS One (2011) 0.93
Defects in the medial entorhinal cortex and dentate gyrus in the mouse model of Sanfilippo syndrome type B. PLoS One (2011) 0.93
Peripheral nerve and neuromuscular junction pathology in Pompe disease. Hum Mol Genet (2014) 0.87
Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice. Gene Ther (2013) 0.86
Therapies of mucopolysaccharidosis IVA (Morquio A syndrome). Expert Opin Orphan Drugs (2013) 0.85
Differential distribution of heparan sulfate glycoforms and elevated expression of heparan sulfate biosynthetic enzyme genes in the brain of mucopolysaccharidosis IIIB mice. Metab Brain Dis (2011) 0.84
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases. Hum Gene Ther (2016) 0.84
Mucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10. Gene Ther (2015) 0.82
Peripheral nervous system neuropathology and progressive sensory impairments in a mouse model of Mucopolysaccharidosis IIIB. PLoS One (2012) 0.81
Gene therapy for neurologic manifestations of mucopolysaccharidoses. Expert Opin Drug Deliv (2014) 0.79
Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts. Biochem J (2014) 0.79
Intracisternal delivery of NFκB-inducible scAAV2/9 reveals locoregional neuroinflammation induced by systemic kainic acid treatment. Front Mol Neurosci (2014) 0.75
Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice. Behav Brain Res (2016) 0.75
Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr (2004) 3.86
Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome). Pediatrics (2008) 2.61
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. Pediatrics (2009) 2.44
The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I. Mol Genet Metab (2007) 2.23
Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain. Mol Ther (2003) 1.94
Innate and adaptive immune activation in the brain of MPS IIIB mouse model. J Neurosci Res (2009) 1.90
Initial report from the Hunter Outcome Survey. Genet Med (2008) 1.80
AAV hybrid serotypes: improved vectors for gene delivery. Curr Gene Ther (2005) 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
Brain MRI findings in patients with mucopolysaccharidosis types I and II and mild clinical presentation. Neuroradiology (2004) 1.61
Host cell DNA repair pathways in adeno-associated viral genome processing. J Virol (2006) 1.43
MCP-1 contributes to arteriovenous fistula failure. J Am Soc Nephrol (2010) 1.23
Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity. Mol Ther (2012) 1.19
Effects of adeno-associated virus DNA hairpin structure on recombination. J Virol (2005) 1.15
Wear of crosslinked polyethylene under different tribological conditions. J Mater Sci Mater Med (2006) 1.12
Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery. Mol Ther (2002) 1.12
Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats. Mol Pain (2006) 1.11
Human alpha defensin 5 expression in the human kidney and urinary tract. PLoS One (2012) 1.08
Safety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy. Mol Ther (2011) 1.04
Respiratory manifestations in mucopolysaccharidoses. Paediatr Respir Rev (2010) 0.99
Development and characterization of novel empty adenovirus capsids and their impact on cellular gene expression. J Virol (2003) 0.98
Mucopolysaccharidosis IIIB, a lysosomal storage disease, triggers a pathogenic CNS autoimmune response. J Neuroinflammation (2010) 0.97
Lentivirus vector purification using anion exchange HPLC leads to improved gene transfer. Biotechniques (2003) 0.96
Preclinical dose ranging studies for enzyme replacement therapy with idursulfase in a knock-out mouse model of MPS II. Mol Genet Metab (2007) 0.95
Idiopathic gingival hyperplasia: a case report with a 17-year followup. Case Rep Dent (2011) 0.95
Differential effects of DNA double-strand break repair pathways on single-strand and self-complementary adeno-associated virus vector genomes. J Virol (2010) 0.95
Adeno-associated virus serotypes 1 to 5 mediated tumor cell directed gene transfer and improvement of transduction efficiency. J Gene Med (2005) 0.94
Evaluation of disease severity in mucopolysaccharidoses. J Pediatr Rehabil Med (2010) 0.93
Bronchoscopy and airway management in patients with mucopolysaccharidoses (MPS). Pediatr Pulmonol (2012) 0.92
Reduction of GAG storage in MPS II mouse model following implantation of encapsulated recombinant myoblasts. J Gene Med (2005) 0.92
Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates. Hum Gene Ther Clin Dev (2014) 0.91
Effect of conformity and contact stress on wear in fixed-bearing total knee prostheses. J Biomech (2009) 0.91
ß-Catenin is markedly induced in a murine model of an arteriovenous fistula: the effect of metalloproteinase inhibition. Am J Physiol Renal Physiol (2010) 0.89
Relationship of sleep to pulmonary function in mucopolysaccharidosis II. J Pediatr (2013) 0.89
Analysis of the interaction between adeno-associated virus and heparan sulfate using atomic force microscopy. Glycobiology (2004) 0.88
Enzyme Replacement Therapy in Mucopolysaccharidosis II Patients Under 1 Year of Age. JIMD Rep (2014) 0.88
Modular preoperative planning software for computer-aided oral implantology and the application of a novel stereolithographic template: a pilot study. Clin Implant Dent Relat Res (2009) 0.84
Differential distribution of heparan sulfate glycoforms and elevated expression of heparan sulfate biosynthetic enzyme genes in the brain of mucopolysaccharidosis IIIB mice. Metab Brain Dis (2011) 0.84
Peripheral nervous system neuropathology and progressive sensory impairments in a mouse model of Mucopolysaccharidosis IIIB. PLoS One (2012) 0.81
Amyloidosis, synucleinopathy, and prion encephalopathy in a neuropathic lysosomal storage disease: the CNS-biomarker potential of peripheral blood. PLoS One (2013) 0.81
The Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Questionnaire: item reduction and further validation. Qual Life Res (2014) 0.79
Longitudinal neurocognitive outcome in an adolescent with Hurler-Scheie syndrome. Neuropsychiatr Dis Treat (2006) 0.78
Glutaric acidemia type 1 in patients of Lumbee heritage from North Carolina. Mol Genet Metab (2006) 0.76
Adult-Onset Presentation of Glutaric Acidemia Type II With Myopathy. J Clin Neuromuscul Dis (2003) 0.76
Effective Depletion of Pre-existing Anti-AAV Antibodies Requires Broad Immune Targeting. Mol Ther Methods Clin Dev (2017) 0.75
Fluorination effects on the shapes of complexes of water with ethers: a rotational study of trifluoroanisole-water. J Phys Chem A (2014) 0.75
The implementation of an integrated computer-aided system for dental implantology. Conf Proc IEEE Eng Med Biol Soc (2008) 0.75
Interplay of phenol and isopropyl isomerism in propofol from broadband chirped-pulse microwave spectroscopy. J Am Chem Soc (2010) 0.75
Lysosomal Disease Network's "WORLD Symposium 2009". Introduction. Mol Genet Metab (2008) 0.75