| scholarly article | Q13442814 |
| P6179 | Dimensions Publication ID | 1025674342 |
| P356 | DOI | 10.1038/GT.2009.85 |
| P932 | PMC publication ID | 4289609 |
| P698 | PubMed publication ID | 19587708 |
| P5875 | ResearchGate publication ID | 26656900 |
| P2093 | author name string | H Fu | |
| D M McCarty | |||
| J Muenzer | |||
| J DiRosario | |||
| K Gulaid | |||
| P2860 | cites work | Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding alpha-N-acetylglucosaminidase | Q24647066 |
| Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis | Q28213353 | ||
| Social interaction and sensorimotor gating abnormalities in mice lacking Dvl1 | Q28595044 | ||
| Expression and characterization of human recombinant and alpha-N-acetylglucosaminidase | Q32125136 | ||
| Osmotic opening of the blood-brain barrier: principles, mechanism, and therapeutic applications | Q33851310 | ||
| Molecular biology of the blood-brain barrier | Q33986161 | ||
| Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood- brain barrier | Q34026516 | ||
| Synergistic effect of cold mannitol and Na(+)/Ca(2+) exchange blocker on blood-brain barrier opening | Q34521227 | ||
| Enzyme replacement therapy for the mucopolysaccharide storage disorders | Q34628680 | ||
| Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial | Q34640765 | ||
| Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB | Q34762478 | ||
| Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. | Q34778098 | ||
| Enzyme replacement therapy in mucopolysaccharidosis type II (Hunter syndrome): a preliminary report | Q35062068 | ||
| Drug delivery to the central nervous system: a review | Q35205301 | ||
| Lysosomal accumulation of SCMAS (subunit c of mitochondrial ATP synthase) in neurons of the mouse model of mucopolysaccharidosis III B. | Q35842650 | ||
| Osmotic therapy: fact and fiction | Q36262656 | ||
| Targeted delivery across the blood-brain barrier | Q36316725 | ||
| Drug delivery across the blood-brain barrier | Q36320755 | ||
| Molecular Trojan horses for blood-brain barrier drug delivery | Q36535369 | ||
| Pathogenic mechanisms in lysosomal disease: a reappraisal of the role of the lysosome | Q36772970 | ||
| Treatment of lysosomal storage disorders : progress with enzyme replacement therapy | Q37025890 | ||
| Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease. | Q37181003 | ||
| Modification of the blood-brain barrier: increased concentration and fate of enzymes entering the brain | Q37312944 | ||
| Innate and adaptive immune activation in the brain of MPS IIIB mouse model. | Q40033388 | ||
| Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector | Q40430846 | ||
| The cryptic life style of adeno-associated virus | Q40524042 | ||
| Cellular and molecular neurosurgery: pathways from concept to reality--part II: vector systems and delivery methodologies for gene therapy of the central nervous system | Q41417789 | ||
| Quantitative correlation between the residual activity of beta-hexosaminidase A and arylsulfatase A and the severity of the resulting lysosomal storage disease | Q41638167 | ||
| Purification and characterization of recombinant human alpha-N-acetylglucosaminidase secreted by Chinese hamster ovary cells | Q41738684 | ||
| Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain | Q42280576 | ||
| Attenuated plasticity in neurons and astrocytes in the mouse model of Sanfilippo syndrome type B. | Q43514109 | ||
| The conjoint importance of the hippocampus and anterior thalamic nuclei for allocentric spatial learning: evidence from a disconnection study in the rat. | Q43732551 | ||
| Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer | Q43747170 | ||
| Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain | Q44686125 | ||
| Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). | Q44883627 | ||
| Advances in the treatment of mucopolysaccharidosis type I. | Q44885185 | ||
| Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders | Q45159507 | ||
| Systemic mannitol-induced hyperosmolality amplifies rAAV2-mediated striatal transduction to a greater extent than local co-infusion | Q45236560 | ||
| Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors. | Q45423330 | ||
| Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum. | Q45552423 | ||
| Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene | Q45628046 | ||
| Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery | Q45734139 | ||
| Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield | Q45747699 | ||
| Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector | Q45769447 | ||
| Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice | Q45867578 | ||
| Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs | Q45874952 | ||
| Prevention of neuropathology in the mouse model of Hurler syndrome | Q45876472 | ||
| Simulation of hematopoiesis: implications for the gene therapy of lysosomal enzyme disorders | Q45877210 | ||
| Determinants of long-term weight maintenance | Q47300155 | ||
| Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice | Q47643319 | ||
| Cytokines, neurotrophins, and oxidative stress in brain disease from mucopolysaccharidosis IIIB. | Q48350856 | ||
| Selective neurodegeneration in murine mucopolysaccharidosis VII is progressive and reversible | Q48439549 | ||
| Gene expression from recombinant viral vectors in the central nervous system after blood-brain barrier disruption. | Q50761827 | ||
| Dimethylmethylene blue-based spectrophotometry of glycosaminoglycans in untreated urine: a rapid screening procedure for mucopolysaccharidoses. | Q50887542 | ||
| Blood-brain barrier disruption using mannitol: time course and electron microscopy studies. | Q50890487 | ||
| Safety and efficacy of a multicenter study using intraarterial chemotherapy in conjunction with osmotic opening of the blood-brain barrier for the treatment of patients with malignant brain tumors. | Q51421065 | ||
| Effect of blood-brain barrier disruption on intact and fragmented monoclonal antibody localization in intracerebral lung carcinoma xenografts. | Q52512571 | ||
| A randomized comparison of intra-arterial versus intravenous BCNU, with or without intravenous 5-fluorouracil, for newly diagnosed patients with malignant glioma | Q55483561 | ||
| Hurler and Hunter syndromes: mutual correction of the defect in cultured fibroblasts | Q68428517 | ||
| Enzyme replacement with recombinant beta-glucuronidase in murine mucopolysaccharidosis type VII: impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival | Q71306595 | ||
| Quantification and characterization of glycosaminoglycans at the nanogram level by a combined azure A-silver staining in agarose gels | Q71666348 | ||
| Percutaneous intravenous injection in neonatal mice | Q77993909 | ||
| P433 | issue | 11 | |
| P921 | main subject | vector-borne disease | Q2083837 |
| P304 | page(s) | 1340-1352 | |
| P577 | publication date | 2009-07-09 | |
| P1433 | published in | Gene Therapy | Q15763095 |
| P1476 | title | Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice | |
| P478 | volume | 16 |
| Q36521756 | A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB. |
| Q45881431 | A GLP-compliant toxicology and biodistribution study: systemic delivery of a rAAV9 vector for the treatment of mucopolysaccharidosis IIIB. |
| Q34214323 | AAV-mediated human PEDF inhibits tumor growth and metastasis in murine colorectal peritoneal carcinomatosis model |
| Q38856794 | Adeno-Associated Virus-Based Gene Therapy for CNS Diseases. |
| Q41633603 | Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice |
| Q42176546 | Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy |
| Q21129328 | Biology of adeno-associated viral vectors in the central nervous system |
| Q39600788 | Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice |
| Q33974900 | Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus-α-L-Iduronidase for Hurler Disease |
| Q42059455 | Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery |
| Q38194635 | Current and potential therapeutic strategies for mucopolysaccharidoses |
| Q34077919 | Defects in the medial entorhinal cortex and dentate gyrus in the mouse model of Sanfilippo syndrome type B. |
| Q42725476 | Detection of amyloid plaques targeted by USPIO-Aβ1-42 in Alzheimer's disease transgenic mice using magnetic resonance microimaging |
| Q34611770 | Detection of amyloid plaques targeted by bifunctional USPIO in Alzheimer's disease transgenic mice using magnetic resonance microimaging |
| Q34757062 | Differential distribution of heparan sulfate glycoforms and elevated expression of heparan sulfate biosynthetic enzyme genes in the brain of mucopolysaccharidosis IIIB mice |
| Q33521174 | Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB). |
| Q43106648 | Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice. |
| Q36235003 | Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II |
| Q30497589 | Expansive Gene Transfer in the Rat CNS Rapidly Produces Amyotrophic Lateral Sclerosis Relevant Sequelae When TDP-43 is Overexpressed |
| Q45863181 | Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating MPS IIIB: Toxicology, Bio-distribution and Immunological Assessments in Primates |
| Q42225564 | Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates |
| Q33917592 | Gene delivery with viral vectors for cerebrovascular diseases |
| Q37903420 | Genetic strategies to study TDP-43 in rodents and to develop preclinical therapeutics for amyotrophic lateral sclerosis |
| Q64054990 | Identification of variable lymphocyte receptors that can target therapeutics to pathologically exposed brain extracellular matrix |
| Q37714337 | Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts |
| Q34714906 | Intra-arterial delivery of AAV vectors to the mouse brain after mannitol mediated blood brain barrier disruption |
| Q34608390 | Intracisternal delivery of NFκB-inducible scAAV2/9 reveals locoregional neuroinflammation induced by systemic kainic acid treatment. |
| Q37696030 | Intrajugular vein delivery of AAV9-RNAi prevents neuropathological changes and weight loss in Huntington's disease mice. |
| Q38603017 | Intravenous rAAV2/9 injection for murine cochlear gene delivery. |
| Q37744753 | Lessons learnt from animal models: pathophysiology of neuropathic lysosomal storage disorders. |
| Q35707046 | Mannitol-facilitated perfusion staining with 2,3,5-triphenyltetrazolium chloride (TTC) for detection of experimental cerebral infarction and biochemical analysis |
| Q27312829 | Monocyte Trafficking, Engraftment, and Delivery of Nanoparticles and an Exogenous Gene into the Acutely Inflamed Brain Tissue - Evaluations on Monocyte-Based Delivery System for the Central Nervous System |
| Q36630212 | Myeloperoxidase Nuclear Imaging for Epileptogenesis |
| Q34438623 | Peripheral nervous system neuropathology and progressive sensory impairments in a mouse model of Mucopolysaccharidosis IIIB. |
| Q35086714 | Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates |
| Q90190759 | Salinomycin-Loaded Iron Oxide Nanoparticles for Glioblastoma Therapy |
| Q45857027 | Serum MIP-1 alpha level: a biomarker for the follow-up of lentiviral therapy in mucopolysaccharidosis IIIB mice |
| Q33695674 | Systemic gene delivery to the central nervous system using Adeno-associated virus |
| Q58765137 | Targeting Root Cause by Systemic scAAV9-h Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice |
| Q38004089 | The advent of AAV9 expands applications for brain and spinal cord gene delivery |
| Q91131848 | Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier |
| Q37687420 | Viral vector-mediated gene transfer for CNS disease |
| Q38746238 | Viral vectors for therapy of neurologic diseases |
| Q35690999 | β2 Agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine Pompe disease |
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