Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A

Extensive transduction and enhanced spread of a modified AAV2 capsid in the non-human primate central nervous system

(2018) Jerusha Naidoo et al.   MOLECULAR THERAPY

Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates

(2018) Kousaku Ohno et al.   Molecular Therapy-Methods & Clinical Development

The Paravascular Pathway for Brain Waste Clearance: Current Understanding, Significance and Controversy

(2017) Andrew Bacyinski et al.   Frontiers in Neuroanatomy

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

(2016) Michaël Hocquemiller et al.   HUMAN GENE THERAPY

Sphingomyelin-induced inhibition of the plasma membrane calcium ATPase causes neurodegeneration in type A Niemann–Pick disease

(2016) A Pérez-Cañamás et al.   MOLECULAR PSYCHIATRY

Quantitative imaging of tissue sections using infrared scanning technology

(2015) Samantha L. Eaton et al.   JOURNAL OF ANATOMY

Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency

(2015) Melissa P. Wasserstein et al.   MOLECULAR GENETICS AND METABOLISM

AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease

(2015) Martin L. Katz et al.   Science Translational Medicine

High sphingomyelin levels induce lysosomal damage and autophagy dysfunction in Niemann Pick disease type A

(2014) E Gabandé-Rodríguez et al.   CELL DEATH AND DIFFERENTIATION

Pharmacological reversion of sphingomyelin-induced dendritic spine anomalies in a Niemann Pick disease type A mouse model

(2014) Ana I Arroyo et al.   EMBO Molecular Medicine

Strong Cortical and Spinal Cord Transduction After AAV7 and AAV9 Delivery into the Cerebrospinal Fluid of Nonhuman Primates

(2013) Lluis Samaranch et al.   HUMAN GENE THERAPY

AAV9-mediated Expression of a Non-self Protein in Nonhuman Primate Central Nervous System Triggers Widespread Neuroinflammation Driven by Antigen-presenting Cell Transduction

(2013) Lluis Samaranch et al.   MOLECULAR THERAPY

Safety Study of Adeno-Associated Virus Serotype 2-Mediated Human Acid Sphingomyelinase Expression in the Nonhuman Primate Brain

(2012) Ernesto A. Salegio et al.   HUMAN GENE THERAPY

Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A

(2012) Jie Bu et al.   MOLECULAR THERAPY

Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusion

(2011) Robin J. Ziegler et al.   EXPERIMENTAL NEUROLOGY

Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

(2011) Lluis Samaranch et al.   HUMAN GENE THERAPY

Brain pathology in Niemann Pick disease type A: insights from the acid sphingomyelinase knockout mice

(2011) Maria Dolores Ledesma et al.   JOURNAL OF NEUROCHEMISTRY

Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates

(2011) Steven J Gray et al.   MOLECULAR THERAPY

Magnetic Resonance Imaging-Guided Delivery of Adeno-Associated Virus Type 2 to the Primate Brain for the Treatment of Lysosomal Storage Disorders

(2010) E. Aguilar Salegio et al.   HUMAN GENE THERAPY

Subcellular Origin of Sphingosine 1-Phosphate Is Essential for Its Toxic Effect in Lyase-deficient Neurons

(2009) Nadine Hagen et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Caffeine and an adenosine A2Areceptor antagonist prevent memory impairment and synaptotoxicity in adult rats triggered by a convulsive episode in early life

(2009) Giana P. Cognato et al.   JOURNAL OF NEUROCHEMISTRY

A Simplified Baculovirus-AAV Expression Vector System Coupled With One-step Affinity Purification Yields High-titer rAAV Stocks From Insect Cells

(2009) Richard H Smith et al.   MOLECULAR THERAPY

Synaptic Vesicle Docking: Sphingosine Regulates Syntaxin1 Interaction with Munc18

(2009) Paola G. Camoletto et al.   PLoS One

Neuropathology of the acid sphingomyelinase knockout mouse model of Niemann-Pick A disease including structure–function studies associated with cerebellar Purkinje cell degeneration

(2008) Shannon L. Macauley et al.   EXPERIMENTAL NEUROLOGY

Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann–Pick A disease

(2008) James C. Dodge et al.   EXPERIMENTAL NEUROLOGY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Ceramide accumulation mediates inflammation, cell death and infection susceptibility in cystic fibrosis

(2008) Volker Teichgräber et al.   NATURE MEDICINE

Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium

(2008) Emyr Lloyd-Evans et al.   NATURE MEDICINE

Anomalous Surface Distribution of Glycosyl Phosphatidyl Inositol–anchored Proteins in Neurons Lacking Acid Sphingomyelinase

(2007) Cristian Galvan et al.   MOLECULAR BIOLOGY OF THE CELL