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CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy.
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- Journal of Clinical Investigation, 2010, v. 120, n. 4, p. 1253, doi. 10.1172/JCI41615
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- Article
Improved Cationic Lipid Formulations for In Vivo Gene Therapy.
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- Annals of the New York Academy of Sciences, 1995, v. 772, n. 1, p. 126, doi. 10.1111/j.1749-6632.1995.tb44738.x
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- Article
Reducing glycosphingolipid biosynthesis in airway cells partially ameliorates disease manifestations in a mouse model of asthma.
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- International Immunology, 2010, v. 22, n. 7, p. 593, doi. 10.1093/intimm/dxq044
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- Article
CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression.
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- Nature Biotechnology, 2008, v. 26, n. 5, p. 549, doi. 10.1038/nbt1399
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- Article
GBA2-Encoded β-Glucosidase Activity Is Involved in the Inflammatory Response to <i>Pseudomonas aeruginosa</i>.
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- PLoS ONE, 2014, v. 9, n. 8, p. 1, doi. 10.1371/journal.pone.0104763
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- Article
A Bispecific Protein Capable of Engaging CTLA-4 and MHCII Protects Non-Obese Diabetic Mice from Autoimmune Diabetes
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- PLoS ONE, 2013, v. 8, n. 5, p. 1, doi. 10.1371/journal.pone.0063530
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- Article
Dysregulation of Multiple Facets of Glycogen Metabolism in a Murine Model of Pompe Disease.
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- PLoS ONE, 2013, v. 8, n. 2, p. 1, doi. 10.1371/journal.pone.0056181
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- Article
Systemic Delivery of a Glucosylceramide Synthase Inhibitor Reduces CNS Substrates and Increases Lifespan in a Mouse Model of Type 2 Gaucher Disease.
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- PLoS ONE, 2012, v. 7, n. 8, p. 1, doi. 10.1371/journal.pone.0043310
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- Article
Proteasome Inhibition Is Partially Effective in Attenuating Pre-Existing Immunity against Recombinant Adeno-Associated Viral Vectors.
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- PLoS ONE, 2012, v. 7, n. 4, p. 1, doi. 10.1371/journal.pone.0034684
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- Article
Iminosugar-Based Inhibitors of Glucosylceramide Synthase Increase Brain Glycosphingolipids and Survival in a Mouse Model of Sandhoff Disease.
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- PLoS ONE, 2011, v. 6, n. 6, p. 1, doi. 10.1371/journal.pone.0021758
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- Article
Comparative Analysis of Acid Sphingomyelinase Distribution in the CNS of Rats and Mice Following Intracerebroventricular Delivery.
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- PLoS ONE, 2011, v. 6, n. 1, p. 1, doi. 10.1371/journal.pone.0016313
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- Article
Substrate Reduction Augments the Efficacy of Enzyme Therapy in a Mouse Model of Fabry Disease.
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- PLoS ONE, 2010, v. 5, n. 11, p. 1, doi. 10.1371/journal.pone.0015033
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- Article
Increased Hepatic Insulin Action in Diet-Induced Obese Mice Following Inhibition of Glucosylceramide Synthase.
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- PLoS ONE, 2010, v. 5, n. 6, p. 1, doi. 10.1371/journal.pone.0011239
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- Article
Mutant GBA1 Expression and Synucleinopathy Risk: First Insights from Cellular and Mouse Models.
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- Neurodegenerative Diseases, 2012, v. 10, n. 1-4, p. 195, doi. 10.1159/000335038
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- Article
Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo.
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- Respiratory Research, 2006, v. 7, p. 26, doi. 10.1186/1465-9921-7-26
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- Article
Characterization of the oligosaccharide structures associated with the cystic fibrosis transmembrane conductance regulator.
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- Glycobiology, 2000, v. 10, n. 11, p. 1225, doi. 10.1093/glycob/10.11.1225
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- Article
Adenovirus-mediated expression of p35 prevents hypoxia/reoxygenation injury by reducing reactive oxygen species and caspase activity
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- Cardiovascular Research, 2002, v. 55, n. 2, p. 309, doi. 10.1016/S0008-6363(02)00412-1
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- Article
Fas ligand/Fas-mediated apoptosis in human coronary artery smooth muscle cells: therapeutic implications of fratricidal mode of action.
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- Cardiovascular Research, 2001, v. 51, n. 4, p. 749, doi. 10.1016/S0008-6363(01)00329-7
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- Article
Intracranial Delivery of CLN2 Reduces Brain Pathology in a Mouse Model of Classical Late Infantile Neuronal Ceroid Lipofuscinosis.
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- Journal of Neuroscience, 2006, v. 26, n. 5, p. 1334, doi. 10.1523/JNEUROSCI.2676-05.2006
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- Article
BK virus infection of the human urinary tract.
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- Journal of Medical Virology, 1993, v. 41, n. 4, p. 301, doi. 10.1002/jmv.1890410408
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- Article
Systemic Administration of a Recombinant AAV1 Vector Encoding IGF-1 Improves Disease Manifestations in SMA Mice.
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- Molecular Therapy, 2014, v. 22, n. 8, p. 1450, doi. 10.1038/mt.2014.84
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- Article
Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy.
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- Molecular Therapy, 2013, v. 21, n. 7, p. 1306, doi. 10.1038/mt.2013.86
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- Article
Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A.
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- Molecular Therapy, 2012, v. 20, n. 10, p. 1893, doi. 10.1038/mt.2012.118
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- Article
Gene Transfer to the CNS Is Efficacious in Immune-primed Mice Harboring Physiologically Relevant Titers of Anti-AAV Antibodies.
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- Molecular Therapy, 2012, v. 20, n. 9, p. 1713, doi. 10.1038/mt.2012.114
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- Article
Gene Transfer Corrects Acute GM2 Gangliosidosis-Potential Therapeutic Contribution of Perivascular Enzyme Flow.
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- Molecular Therapy, 2012, v. 20, n. 8, p. 1489, doi. 10.1038/mt.2012.44
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- Article
Disease progression in a mouse model of amyotrophic lateral sclerosis: the influence of chronic stress and corticosterone.
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- FASEB Journal, 2011, v. 25, n. 12, p. 4369, doi. 10.1096/fj.11-190819
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- Article
Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system.
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- FASEB Journal, 2011, v. 25, n. 10, p. 3505, doi. 10.1096/fj.11-182311
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- Article
Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A.
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- Science Translational Medicine, 2019, v. 11, n. 506, p. 1, doi. 10.1126/scitranslmed.aat3738
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- Article
Acid β-glucosidase mutants linked to gaucher disease, parkinson disease, and lewy body dementia alter α-synuclein processing.
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- Annals of Neurology, 2011, v. 69, n. 6, p. 940, doi. 10.1002/ana.22400
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- Article
Substrate reduction therapy using Genz‐667161 reduces levels of pathogenic components in a mouse model of neuronopathic forms of Gaucher disease.
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- Journal of Neurochemistry, 2021, v. 156, n. 5, p. 692, doi. 10.1111/jnc.15136
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- Article
Low-frequency ultrasound increases non-viral gene transfer to the mouse lung.
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- Acta Biochimica et Biophysica Sinica, 2010, v. 42, n. 1, p. 45, doi. 10.1093/abbs/gmp100
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- Article
AAV‐mediated expression of galactose‐1‐phosphate uridyltransferase corrects defects of galactose metabolism in classic galactosemia patient fibroblasts.
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- Journal of Inherited Metabolic Disease, 2022, v. 45, n. 3, p. 481, doi. 10.1002/jimd.12468
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- Article
Priapism in a Fabry disease mouse model is associated with upregulated penile nNOS and eNOS expression.
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- Journal of Inherited Metabolic Disease, 2018, v. 41, n. 2, p. 231, doi. 10.1007/s10545-017-0107-6
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- Article
Tetrahydrobiopterin deficiency in the pathogenesis of Fabry disease.
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- Human Molecular Genetics, 2017, v. 26, n. 6, p. 1182, doi. 10.1093/hmg/ddx032
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- Article
Glucocerebrosidase modulates cognitive and motor activities in murine models of Parkinson's disease.
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- Human Molecular Genetics, 2016, v. 25, n. 13, p. 2645, doi. 10.1093/hmg/ddw124
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- Article
Reversibility of neuropathology in Tay–Sachs-related diseases.
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- Human Molecular Genetics, 2014, v. 23, n. 3, p. 730
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- Article
Marked differences in neurochemistry and aggregates despite similar behavioural and neuropathological features of Huntington disease in the full-length BACHD and YAC128 mice.
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- Human Molecular Genetics, 2012, v. 21, n. 10, p. 2219, doi. 10.1093/hmg/dds037
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- Article
Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain.
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- Human Molecular Genetics, 2011, v. 20, n. 22, p. 4371, doi. 10.1093/hmg/ddr364
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- Article
Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy.
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- Science Translational Medicine, 2011, v. 3, n. 72, p. 1, doi. 10.1126/scitranslmed.3001777
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- Article
Inhibiting Glycosphingolipid Synthesis Improves Glycemic Control and Insulin Sensitivity in Animal Models of Type 2 Diabetes.
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- Diabetes, 2007, v. 56, n. 5, p. 1210, doi. 10.2337/db06-0719
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- Article
Polyamine Analogues of 3 β-[ N-( N′, N′-Dimethylaminoethane)carbamoyl]cholesterol (DC-Chol) as Agents for Gene Delivery.
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- Chemistry - A European Journal, 1998, v. 4, n. 1, p. 137, doi. 10.1002/(SICI)1521-3765(199801)4:1<137::AID-CHEM137>3.0.CO;2-2
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- Article
Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease.
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- Molecular Medicine, 2015, v. 21, n. 1, p. 389, doi. 10.2119/molmed.2015.00088
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- Article
Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium.
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- Journal of Gene Medicine, 2012, v. 14, n. 7, p. 491, doi. 10.1002/jgm.2643
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- Article
Adeno-associated virus-mediated expression of acid sphingomyelinase decreases atherosclerotic lesion formation in apolipoprotein E.
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- Journal of Gene Medicine, 2011, v. 13, n. 6, p. 324, doi. 10.1002/jgm.1575
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- Article
Detection of CFTR transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavity.
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- Journal of Gene Medicine, 2010, v. 12, n. 1, p. 55, doi. 10.1002/jgm.1413
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- Article
A hypoxic inducible factor-1α hybrid enhances collateral development and reduces vascular leakage in diabetic rats.
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- Journal of Gene Medicine, 2009, v. 11, n. 5, p. 390, doi. 10.1002/jgm.1318
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- Article
Acute intermittent porphyria: vector optimization for gene therapy.
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- Journal of Gene Medicine, 2007, v. 9, n. 9, p. 806, doi. 10.1002/jgm.1074
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- Article
Electroporation enhances reporter gene expression following delivery of naked plasmid DNA to the lung.
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- Journal of Gene Medicine, 2007, v. 9, n. 5, p. 369, doi. 10.1002/jgm.1026
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- Article
Identification of transfected cell types following non-viral gene transfer to the murine lung.
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- Journal of Gene Medicine, 2007, v. 9, n. 3, p. 184, doi. 10.1002/jgm.1007
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- Article
AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease.
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- Journal of Gene Medicine, 2006, v. 8, n. 6, p. 719, doi. 10.1002/jgm.901
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- Article