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Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity

EMBO molecular medicine, 2022-01, Vol.14 (1), p.e13968-n/a

2022

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Cerebellar Pathology in an Inducible Mouse Model of Friedreich Ataxia

Frontiers in neuroscience, 2022-03, Vol.16, p.819569

2022

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The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy

Molecular therapy. Methods & clinical development, 2019-06, Vol.13, p.440-452

2019

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CNS Adverse Effects: From Functional Observation Battery/Irwin Tests to Electrophysiology

Handbook of experimental pharmacology, 2015, Vol.229, p.83

2015

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Human Mesenchymal Stromal Cells and their Derivative, SB623 Cells, Rescue Neural Cells via Trophic Support following in Vitro Ischemia

Cell transplantation, 2010-01, Vol.19 (8), p.973-984

2010

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AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich’s ataxia

Molecular therapy. Methods & clinical development, 2024-03, Vol.32 (1), p.101193-101193, Article 101193

2024

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Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood

Molecular therapy. Methods & clinical development, 2022-09, Vol.26, p.519-531

2022

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Mice expressing the ADNFLE valine 287 leucine mutation of the Β2 nicotinic acetylcholine receptor subunit display increased sensitivity to acute nicotine administration and altered presynaptic nicotinic receptor function

Pharmacology, biochemistry and behavior, 2013-01, Vol.103 (3), p.603-621

2013

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Nicotine-Induced Dystonic Arousal Complex in a Mouse Line Harboring a Human Autosomal-Dominant Nocturnal Frontal Lobe Epilepsy Mutation

The Journal of neuroscience, 2007-09, Vol.27 (38), p.10128-10142

2007

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Point Mutant Mice with Hypersensitive α4 Nicotinic Receptors Show Dopaminergic Deficits and Increased Anxiety

Proceedings of the National Academy of Sciences - PNAS, 2001-02, Vol.98 (5), p.2786-2791

2001

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Demonstration of functional α4-containing nicotinic receptors in the medial habenula

Neuropharmacology, 2009-01, Vol.56 (1), p.247-253

2009

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Toxic effects of MPP + and MPTP in PC12 cells independent of reactive oxygen species formation

Brain research, 2001-06, Vol.905 (1), p.199-206

2001

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Increased Sensitivity to Agonist-Induced Seizures, Straub Tail, and Hippocampal Theta Rhythm in Knock-In Mice Carrying Hypersensitive α4 Nicotinic Receptors

The Journal of neuroscience, 2003-04, Vol.23 (7), p.2582-2590

2003

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Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy

Gene therapy, 2023-08, Vol.30 (7-8), p.581-586

2023

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Ex Vivo Gene Therapy: Graft-versus-host Disease (GVHD) in NSG™ (NOD.Cg-Prkdcscid Il2rgtm1Wjl/SzJ) Mice Transplanted with CD34+ Human Hematopoietic Stem Cells

Toxicologic pathology, 2019-07, Vol.47 (5), p.656-660

2019

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Safety Findings of Dosing Gene Therapy Vectors in NHP With Pre-existing or Treatment-Emergent Anti-capsid Antibodies

Toxicologic pathology, 2023-07, Vol.51 (5), p.246-256

2023

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Transgenic Mice Neuronally Expressing Baculoviral p35 are Resistant to Diverse Types of Induced Apoptosis, including Seizure-Associated Neurodegeneration

Proceedings of the National Academy of Sciences - PNAS, 2000-02, Vol.97 (5), p.2270-2275

2000

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Impact of Pre-Existing Immunogenicity to AAV on Vector Transduction By Bmn 270, an AAV5-Based Gene Therapy Treatment for Hemophilia A

Blood, 2017-12, Vol.130, p.3332-3332

2017

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Approaches to seizure risk assessment in preclinical drug discovery

Drug discovery today, 2009-09, Vol.14 (17), p.876-884

2009

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α4 Nicotinic Receptors in preBötzinger Complex Mediate Cholinergic/Nicotinic Modulation of Respiratory Rhythm

The Journal of neuroscience, 2008-01, Vol.28 (2), p.519-528

2008

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Novel Seizure Phenotype and Sleep Disruptions in Knock-In Mice with Hypersensitive α4 Nicotinic Receptors

The Journal of neuroscience, 2005-12, Vol.25 (49), p.11396-11411

2005

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Rapid reduction of ATP synthesis and lack of free radical formation by MPP + in rat brain synaptosomes and mitochondria

Brain research, 2003-06, Vol.975 (1), p.214-221

2003

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Comparison of the rat and mouse hippocampal slice preparations for the detection of seizure liability

Journal of pharmacological and toxicological methods, 2010-09, Vol.62 (2), p.e21-e21

2010

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Demonstration of functional alpha 4-containing nicotinic receptors in the medial habenula

Neuropharmacology, 2009-01, Vol.56 (1), p.247-253

2009

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Increased Sensitivity to Agonist-Induced Seizures, Straub Tail, and Hippocampal Theta Rhythm in Knock-In Mice Carrying Hypersensitive alpha 4 Nicotinic Receptors

The Journal of neuroscience, 2003-04, Vol.23 (7), p.2582-2590

2003

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Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity

Cerebellar Pathology in an Inducible Mouse Model of Friedreich Ataxia

The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy

CNS Adverse Effects: From Functional Observation Battery/Irwin Tests to Electrophysiology

Human Mesenchymal Stromal Cells and their Derivative, SB623 Cells, Rescue Neural Cells via Trophic Support following in Vitro Ischemia

AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich’s ataxia

Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood

Mice expressing the ADNFLE valine 287 leucine mutation of the Β2 nicotinic acetylcholine receptor subunit display increased sensitivity to acute nicotine administration and altered presynaptic nicotinic receptor function

Nicotine-Induced Dystonic Arousal Complex in a Mouse Line Harboring a Human Autosomal-Dominant Nocturnal Frontal Lobe Epilepsy Mutation

Point Mutant Mice with Hypersensitive α4 Nicotinic Receptors Show Dopaminergic Deficits and Increased Anxiety

Demonstration of functional α4-containing nicotinic receptors in the medial habenula

Toxic effects of MPP + and MPTP in PC12 cells independent of reactive oxygen species formation

Increased Sensitivity to Agonist-Induced Seizures, Straub Tail, and Hippocampal Theta Rhythm in Knock-In Mice Carrying Hypersensitive α4 Nicotinic Receptors

Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy

Ex Vivo Gene Therapy: Graft-versus-host Disease (GVHD) in NSG™ (NOD.Cg-Prkdcscid Il2rgtm1Wjl/SzJ) Mice Transplanted with CD34+ Human Hematopoietic Stem Cells

Safety Findings of Dosing Gene Therapy Vectors in NHP With Pre-existing or Treatment-Emergent Anti-capsid Antibodies

Transgenic Mice Neuronally Expressing Baculoviral p35 are Resistant to Diverse Types of Induced Apoptosis, including Seizure-Associated Neurodegeneration

Impact of Pre-Existing Immunogenicity to AAV on Vector Transduction By Bmn 270, an AAV5-Based Gene Therapy Treatment for Hemophilia A

Approaches to seizure risk assessment in preclinical drug discovery

α4 Nicotinic Receptors in preBötzinger Complex Mediate Cholinergic/Nicotinic Modulation of Respiratory Rhythm

Novel Seizure Phenotype and Sleep Disruptions in Knock-In Mice with Hypersensitive α4 Nicotinic Receptors

Rapid reduction of ATP synthesis and lack of free radical formation by MPP + in rat brain synaptosomes and mitochondria

Comparison of the rat and mouse hippocampal slice preparations for the detection of seizure liability

Demonstration of functional alpha 4-containing nicotinic receptors in the medial habenula

Increased Sensitivity to Agonist-Induced Seizures, Straub Tail, and Hippocampal Theta Rhythm in Knock-In Mice Carrying Hypersensitive alpha 4 Nicotinic Receptors

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