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The respiratory neuromuscular system in Pompe disease

Respiratory physiology & neurobiology, 2013-11, Vol.189 (2), p.241-249

2013

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Liposomes to target peripheral neurons and Schwann cells

PloS one, 2013-11, Vol.8 (11), p.e78724

2013

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Sustained Correction of Motoneuron Histopathology Following Intramuscular Delivery of AAV in Pompe Mice

Molecular therapy, 2014-04, Vol.22 (4), p.702-712

2014

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Peripheral nerve and neuromuscular junction pathology in Pompe disease

Human molecular genetics, 2015-02, Vol.24 (3), p.625-636

2015

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Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized NAGLU vector

Human molecular genetics, 2023-01, Vol.32 (3), p.417-430

2023

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Characterization of a transgenic short hairpin RNA-induced murine model of Tafazzin deficiency

Human gene therapy, 2011-07, Vol.22 (7), p.865

2011

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Mechanical Ventilation for Duchenne Muscular Dystrophy: Sinner or Saint?

Muscle & nerve, 2018-03, Vol.57 (3), p.353-355

2018

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Neuropathology in respiratory-related motoneurons in young Pompe ( Gaa−/− ) mice

Respiratory physiology & neurobiology, 2016-06, Vol.227, p.48-55

2016

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Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease

Molecular therapy, 2013-09, Vol.21 (9), p.1661-1667

2013

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Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease

Human gene therapy, 2016-01, Vol.27 (1), p.43

2016

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Reduction of Autophagic Accumulation in Pompe Disease Mouse Model Following Gene Therapy

Current gene therapy, 2019-01, Vol.19 (3), p.197

2019

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Caspase-3 Regulation of Diaphragm Myonuclear Domain during Mechanical Ventilation-induced Atrophy

American journal of respiratory and critical care medicine, 2007-01, Vol.175 (2), p.150-159

2007

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Rapamycin improves peripheral nerve myelination while it fails to benefit neuromuscular performance in neuropathic mice

Neurobiology of disease, 2014-10, Vol.70, p.224-236

2014

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Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease

Annals of neurology, 2015-08, Vol.78 (2), p.222-234

2015

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Xanthine oxidase contributes to mechanical ventilation-induced diaphragmatic oxidative stress and contractile dysfunction

Journal of applied physiology (1985), 2009-02, Vol.106 (2), p.385-394

2009

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Apocynin attenuates diaphragm oxidative stress and protease activation during prolonged mechanical ventilation

Critical care medicine, 2009-04, Vol.37 (4), p.1373-1379

2009

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Locomotor and skeletal muscle abnormalities in trembler J neuropathic mice

Muscle & nerve, 2018-04, Vol.57 (4), p.664-671

2018

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Stimulation of Respiratory Motor Output and Ventilation in a Murine Model of Pompe Disease by Ampakines

American journal of respiratory cell and molecular biology, 2015-09, Vol.53 (3), p.326-335

2015

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A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice

Molecular neurobiology, 2018-06, Vol.55 (6), p.5299-5309

2018

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Gene therapy approaches for lysosomal storage disease: next-generation treatment

Human gene therapy, 2012-08, Vol.23 (8), p.808

2012

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Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy

Annals of translational medicine, 2019-07, Vol.7 (13), p.290-290

2019

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Spinal Delivery of AAV Vector Restores Enzyme Activity and Increases Ventilation in Pompe Mice

Molecular therapy, 2012-01, Vol.20 (1), p.21-27

2012

Volltextzugriff (PDF)

Ischemia-reperfusion-induced calpain activation and SERCA2a degradation are attenuated by exercise training and calpain inhibition

American journal of physiology. Heart and circulatory physiology, 2006-01, Vol.290 (1), p.H128-H136

2006

Volltextzugriff (PDF)

A scalable method for the production of high-titer and high-quality adeno-associated type 9 vectors using the HSV platform

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.16031-16031, Article 16031

2016

Volltextzugriff (PDF)

Pompe disease gene therapy: Gene Therapy

Human molecular genetics, 2011-04, Vol.20 (1), p.R61-R68

2011

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The respiratory neuromuscular system in Pompe disease

Liposomes to target peripheral neurons and Schwann cells

Sustained Correction of Motoneuron Histopathology Following Intramuscular Delivery of AAV in Pompe Mice

Peripheral nerve and neuromuscular junction pathology in Pompe disease

Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized NAGLU vector

Characterization of a transgenic short hairpin RNA-induced murine model of Tafazzin deficiency

Mechanical Ventilation for Duchenne Muscular Dystrophy: Sinner or Saint?

Neuropathology in respiratory-related motoneurons in young Pompe ( Gaa−/− ) mice

Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease

Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease

Reduction of Autophagic Accumulation in Pompe Disease Mouse Model Following Gene Therapy

Caspase-3 Regulation of Diaphragm Myonuclear Domain during Mechanical Ventilation-induced Atrophy

Rapamycin improves peripheral nerve myelination while it fails to benefit neuromuscular performance in neuropathic mice

Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease

Xanthine oxidase contributes to mechanical ventilation-induced diaphragmatic oxidative stress and contractile dysfunction

Apocynin attenuates diaphragm oxidative stress and protease activation during prolonged mechanical ventilation

Locomotor and skeletal muscle abnormalities in trembler J neuropathic mice

Stimulation of Respiratory Motor Output and Ventilation in a Murine Model of Pompe Disease by Ampakines

A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice

Gene therapy approaches for lysosomal storage disease: next-generation treatment

Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy

Spinal Delivery of AAV Vector Restores Enzyme Activity and Increases Ventilation in Pompe Mice

Ischemia-reperfusion-induced calpain activation and SERCA2a degradation are attenuated by exercise training and calpain inhibition

A scalable method for the production of high-titer and high-quality adeno-associated type 9 vectors using the HSV platform

Pompe disease gene therapy: Gene Therapy

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