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Improved Cationic Lipid Formulations for In Vivo Gene Therapy.
- Published in:
- 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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- Publication type:
- Article
Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease.
- Published in:
- Molecular Medicine, 2015, v. 21, n. 1, p. 389, doi. 10.2119/molmed.2015.00088
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- Publication type:
- 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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- Publication type:
- 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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- Publication type:
- 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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- Publication type:
- 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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- Publication type:
- 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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- Publication type:
- Article
Electroporation enhances reporter gene expression following delivery of naked plasmid DNA to the lung.
- Published in:
- Journal of Gene Medicine, 2007, v. 9, n. 5, p. 369, doi. 10.1002/jgm.1026
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- Publication type:
- 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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- Publication type:
- 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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- Publication type:
- Article
Glucosylceramide synthase inhibition reduces ganglioside GM3 accumulation, alleviates amyloid neuropathology, and stabilizes remote contextual memory in a mouse model of Alzheimer's disease.
- Published in:
- Alzheimer's Research & Therapy, 2022, v. 14, n. 1, p. 1, doi. 10.1186/s13195-022-00966-0
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- Publication type:
- Article
Fibrillin-1 Regulates Skeletal Stem Cell Differentiation by Modulating TGFβ Activity Within the Marrow Niche.
- Published in:
- Journal of Bone & Mineral Research, 2016, v. 31, n. 1, p. 86, doi. 10.1002/jbmr.2598
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- Publication type:
- Article
Disease progression in a mouse model of amyotrophic lateral sclerosis: the influence of chronic stress and corticosterone.
- Published in:
- FASEB Journal, 2011, v. 25, n. 12, p. 4369, doi. 10.1096/fj.11-190819
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- Publication type:
- 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.
- Published in:
- FASEB Journal, 2011, v. 25, n. 10, p. 3505, doi. 10.1096/fj.11-182311
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- Publication type:
- Article
Systemic Administration of a Recombinant AAV1 Vector Encoding IGF-1 Improves Disease Manifestations in SMA Mice.
- Published in:
- Molecular Therapy, 2014, v. 22, n. 8, p. 1450, doi. 10.1038/mt.2014.84
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- Publication type:
- Article
Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 7, p. 1306, doi. 10.1038/mt.2013.86
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- Publication type:
- Article
Reversibility of neuropathology in Tay–Sachs-related diseases.
- Published in:
- Human Molecular Genetics, 2014, v. 23, n. 3, p. 730
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- Publication type:
- Article
Marked differences in neurochemistry and aggregates despite similar behavioural and neuropathological features of Huntington disease in the full-length BACHD and YAC128 mice.
- Published in:
- Human Molecular Genetics, 2012, v. 21, n. 10, p. 2219, doi. 10.1093/hmg/dds037
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- Publication type:
- Article
Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain.
- Published in:
- Human Molecular Genetics, 2011, v. 20, n. 22, p. 4371, doi. 10.1093/hmg/ddr364
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- Publication type:
- Article
Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A.
- Published in:
- Science Translational Medicine, 2019, v. 11, n. 506, p. 1, doi. 10.1126/scitranslmed.aat3738
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- Publication type:
- Article
Inhibiting Glycosphingolipid Synthesis Improves Glycemic Control and Insulin Sensitivity in Animal Models of Type 2 Diabetes.
- Published in:
- Diabetes, 2007, v. 56, n. 5, p. 1210, doi. 10.2337/db06-0719
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- Publication type:
- Article
Targeting nuclear RNA for in vivo correction of myotonic dystrophy.
- Published in:
- Nature, 2012, v. 488, n. 7409, p. 111, doi. 10.1038/nature11362
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- Publication type:
- Article
Mutant GBA1 Expression and Synucleinopathy Risk: First Insights from Cellular and Mouse Models.
- Published in:
- Neurodegenerative Diseases, 2012, v. 10, n. 1-4, p. 195, doi. 10.1159/000335038
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- Publication type:
- Article
BK virus infection of the human urinary tract.
- Published in:
- Journal of Medical Virology, 1993, v. 41, n. 4, p. 301, doi. 10.1002/jmv.1890410408
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- Publication type:
- Article
Inhibition of osteoclastogenesis by prolyl hydroxylase inhibitor dimethyloxallyl glycine.
- Published in:
- 2010
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- Publication type:
- journal article
Characterization of the oligosaccharide structures associated with the cystic fibrosis transmembrane conductance regulator.
- Published in:
- Glycobiology, 2000, v. 10, n. 11, p. 1225, doi. 10.1093/glycob/10.11.1225
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- Publication type:
- Article
Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 10, p. 1893, doi. 10.1038/mt.2012.118
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- Publication type:
- Article
Gene Transfer to the CNS Is Efficacious in Immune-primed Mice Harboring Physiologically Relevant Titers of Anti-AAV Antibodies.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 9, p. 1713, doi. 10.1038/mt.2012.114
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- Publication type:
- Article
Gene Transfer Corrects Acute GM2 Gangliosidosis-Potential Therapeutic Contribution of Perivascular Enzyme Flow.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 8, p. 1489, doi. 10.1038/mt.2012.44
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- Publication type:
- Article
Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 11, p. 1999, doi. 10.1038/mt.2011.119
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- Publication type:
- Article
Induction of Immune Tolerance to a Therapeutic Protein by Intrathymic Gene Delivery.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 12, p. 2146, doi. 10.1038/mt.2010.164
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- Publication type:
- Article
AAV4-mediated Expression of IGF-1 and VEGF Within Cellular Components of the Ventricular System Improves Survival Outcome in Familial ALS Mice.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 12, p. 2075, doi. 10.1038/mt.2010.206
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- Publication type:
- Article
Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 11, p. 1983, doi. 10.1038/mt.2010.175
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- Publication type:
- Article
Evaluation of Systemic Follistatin as an Adjuvant to Stimulate Muscle Repair and Improve Motor Function in Pompe Mice.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 9, p. 1584, doi. 10.1038/mt.2010.110
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- Publication type:
- Article
AAV8-mediated Gene Therapy Prevents Induced Biochemical Attacks of Acute Intermittent Porphyria and Improves Neuromotor Function.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 1, p. 17, doi. 10.1038/mt.2009.250
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- Publication type:
- Article
Glycoengineered Acid α-Glucosidase With Improved Efficacy at Correcting the Metabolic Aberrations and Motor Function Deficits in a Mouse Model of Pompe Disease.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 6, p. 954, doi. 10.1038/mt.2009.37
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- Publication type:
- Article
Delivery of AAV-IGF-1 to the CNS Extends Survival in ALS Mice Through Modification of Aberrant Glial Cell Activity.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 6, p. 1056, doi. 10.1038/mt.2008.60
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- Publication type:
- Article
Intraventricular Enzyme Replacement Improves Disease Phenotypes in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 4, p. 649, doi. 10.1038/mt.2008.9
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- Publication type:
- Article
Timing of Therapeutic Intervention Determines Functional and Survival Outcomes in a Mouse Model of Late Infantile Batten Disease.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 10, p. 1782, doi. 10.1038/sj.mt.6300249
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- Publication type:
- Article
Correction of the Biochemical and Functional Deficits in Fabry Mice Following AAV8–mediated Hepatic Expression of α-galactosidase A.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 3, p. 492, doi. 10.1038/sj.mt.6300066
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- Publication type:
- Article
Optimizing Aerosol Gene Delivery and Expression in the Ovine Lung.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 2, p. 348, doi. 10.1038/sj.mt.6300058
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- Publication type:
- Article
893. Second Generation Gene Therapy Vector for Classical Late Infantile Neuronal Ceroid Lipofuscinosis.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S344, doi. 10.1016/j.ymthe.2006.08.982
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- Publication type:
- Article
1062. Intracerebellar Injection of AAV-IGF-1 Improves Motor Function and Extends Survival in a Mouse Model of Amyotrophic Lateral Sclerosis.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S407, doi. 10.1016/j.ymthe.2006.08.1160
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- Publication type:
- Article
2. Identification of Different Modes of Viral Transport in the Non-Human Primate Brain after Convection-Enhanced Delivery of AAV Serotype Vectors.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S1, doi. 10.1016/j.ymthe.2006.08.012
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- Publication type:
- Article
217. Comparison of Hepatic Vein and Peripheral Vein Delivery of an AAV2/8 Vector Encoding Human a-Galactosidase A to Rhesus Macaques.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S84, doi. 10.1016/j.ymthe.2006.08.242
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- Publication type:
- Article
226. Effective Gene Therapy in an Authentic Mouse Model of Tay-Sachs Related Diseases.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S86, doi. 10.1016/j.ymthe.2006.08.251
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- Publication type:
- Article
417. Combination Brain and Systemic Injections of AAV Results in Whole Body Therapy and Extension of Lifespan in the Niemann-Pick Mouse.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S160, doi. 10.1016/j.ymthe.2006.08.481
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- Publication type:
- Article
695. Low-Frequency Ultrasound Increases Non-Viral Lung Gene Transfer.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S269, doi. 10.1016/j.ymthe.2006.08.773
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- Publication type:
- Article
786. Development of Zero-CpG Plasmids with Reduced Inflammatory Responses Following Delivery of Lipid/pDNA Complexes to the Mouse Lung.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S304, doi. 10.1016/j.ymthe.2006.08.873
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- Publication type:
- Article
AAV8-Mediated Hepatic Expression of Acid Sphingomyelinase Corrects the Metabolic Defect in the Visceral Organs of a Mouse Model of Niemann–Pick Disease
- Published in:
- Molecular Therapy, 2005, v. 12, n. 3, p. 431, doi. 10.1016/j.ymthe.2005.03.011
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- Publication type:
- Article