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Aptamer-programmable adeno-associated viral vectors as a novel platform for cell-specific gene transfer

Molecular therapy. Nucleic acids, 2023-03, Vol.31, p.383-397

2023

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AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

Molecular therapy. Methods & clinical development, 2019-03, Vol.12, p.85-101

2019

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1-Butanol dehydration and oxidation over vanadium phosphate catalysts

Applied catalysis. A, General, 2023-07, Vol.661, p.119243, Article 119243

2023

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Targeting Mitochondrial Defects to Increase Longevity in Animal Models of Neurodegenerative Diseases

Advances in experimental medicine and biology, 2019-01, Vol.1134, p.89-110

2019

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SO2 resistant soot oxidation catalysts based on orthovanadates

Catalysis communications, 2017-07, Vol.97, p.120-124

2017

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Autophagy determines efficiency of liver‐directed gene therapy with adeno‐associated viral vectors

Hepatology (Baltimore, Md.), 2017-07, Vol.66 (1), p.252-265

2017

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Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase

Molecular therapy, 2020-09, Vol.28 (9), p.2056-2072

2020

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A New Process for Maleic Anhydride Synthesis from a Renewable Building Block: The Gas-Phase Oxidehydration of Bio-1-butanol

ChemSusChem, 2015-07, Vol.8 (13), p.2250-2259

2015

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Catalytic Biorefining of Ethanol from Wine Waste to Butanol and Higher Alcohols: Modeling the Life Cycle Assessment and Process Design

ACS sustainable chemistry & engineering, 2019-01, Vol.7 (1), p.224-237

2019

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Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase Complex

Molecular therapy, 2021-03, Vol.29 (3), p.1016-1027

2021

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Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase

Molecular therapy, 2020-09, Vol.28 (9), p.2056-2072

2020

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Structure–Reactivity Correlations in Vanadium‐Containing Catalysts for One‐Pot Glycerol Oxidehydration to Acrylic Acid

ChemSusChem, 2017-01, Vol.10 (1), p.234-244

2017

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Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression

Molecular therapy, 2018-03, Vol.26 (3), p.890-901

2018

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Safety and efficacy evaluation of investigational liver gene transfer for secretable GAA in the treatment of Pompe disease

Molecular genetics and metabolism, 2019-02, Vol.126 (2), p.S24-S24

2019

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Liver expression of secretable GAA rescues advanced Pompe disease at the biochemical, functional, and transcriptional level in Gaa−/−mice

Molecular genetics and metabolism, 2020-02, Vol.129 (2), p.S36-S37

2020

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Tandem promoter design confers tolerogenic and persistent transgene expression to AAV gene therapy in neonate Pompe mice

Molecular genetics and metabolism, 2019-02, Vol.126 (2), p.S41-S41

2019

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Functional, biochemical and transcriptional rescue of advanced Pompe disease in mice with liver expression of secretable GAA

Molecular genetics and metabolism, 2019-02, Vol.126 (2), p.S35-S35

2019

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Preclinical development of SPK-3006, an investigational liver-directed AAV gene therapy for the treatment of Pompe disease

Molecular genetics and metabolism, 2020-02, Vol.129 (2), p.S22-S23

2020

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Preclinical development of , an investigational liver-directed AAV gene therapy for the treatment of Pompe disease

Molecular genetics and metabolism, 2020-02, Vol.129 (2), p.S22-S23

2020

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Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression

Molecular therapy, 2018-03, Vol.26 (3), p.890-901

2018

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Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

Scientific reports, 2016-11, Vol.6 (1), p.36182-36182, Article 36182

2016

Volltextzugriff (PDF)

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

Molecular therapy. Methods & clinical development, 2019-03, Vol.12, p.85-101

2019

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Moro orange juice prevents fatty liver in mice

World journal of gastroenterology : WJG, 2012-08, Vol.18 (29), p.3862-3868

2012

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Stacking the Nanochemistry Deck: Structural and Compositional Diversity in One-Dimensional Photonic Crystals

Advanced materials (Weinheim), 2009-04, Vol.21 (16), p.1641-1646

2009

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Nanofabrication by self-assembly

Materials today (Kidlington, England), 2009-05, Vol.12 (5), p.12-23

2009

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Aptamer-programmable adeno-associated viral vectors as a novel platform for cell-specific gene transfer

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

1-Butanol dehydration and oxidation over vanadium phosphate catalysts

Targeting Mitochondrial Defects to Increase Longevity in Animal Models of Neurodegenerative Diseases

SO2 resistant soot oxidation catalysts based on orthovanadates

Autophagy determines efficiency of liver‐directed gene therapy with adeno‐associated viral vectors

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase

A New Process for Maleic Anhydride Synthesis from a Renewable Building Block: The Gas-Phase Oxidehydration of Bio-1-butanol

Catalytic Biorefining of Ethanol from Wine Waste to Butanol and Higher Alcohols: Modeling the Life Cycle Assessment and Process Design

Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase Complex

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase

Structure–Reactivity Correlations in Vanadium‐Containing Catalysts for One‐Pot Glycerol Oxidehydration to Acrylic Acid

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression

Safety and efficacy evaluation of investigational liver gene transfer for secretable GAA in the treatment of Pompe disease

Liver expression of secretable GAA rescues advanced Pompe disease at the biochemical, functional, and transcriptional level in Gaa−/−mice

Tandem promoter design confers tolerogenic and persistent transgene expression to AAV gene therapy in neonate Pompe mice

Functional, biochemical and transcriptional rescue of advanced Pompe disease in mice with liver expression of secretable GAA

Preclinical development of SPK-3006, an investigational liver-directed AAV gene therapy for the treatment of Pompe disease

Preclinical development of , an investigational liver-directed AAV gene therapy for the treatment of Pompe disease

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

Moro orange juice prevents fatty liver in mice

Stacking the Nanochemistry Deck: Structural and Compositional Diversity in One-Dimensional Photonic Crystals

Nanofabrication by self-assembly

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