Lysosomal and network alterations in human mucopolysaccharidosis type VII iPSC-derived neurons

Axonal transport and maturation of lysosomes

(2018) Shawn M Ferguson   CURRENT OPINION IN NEUROBIOLOGY

Transdifferentiation of human adult peripheral blood T cells into neurons

(2018) Koji Tanabe et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Clinical course of sly syndrome (mucopolysaccharidosis type VII)

(2016) Adriana M Montaño et al.   JOURNAL OF MEDICAL GENETICS

Integrated analysis of proteome and transcriptome changes in the mucopolysaccharidosis type VII mouse hippocampus

(2016) Michael K. Parente et al.   MOLECULAR GENETICS AND METABOLISM

Astrocytes and lysosomal storage diseases

(2016) K.V. Rama Rao et al.   NEUROSCIENCE

Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy

(2016) Vasco Meneghini et al.   Stem Cells Translational Medicine

Mucopolysaccharidosis VII in a Cat Caused by 2 Adjacent Missense Mutations in theGUSBGene

(2015) P. Wang et al.   JOURNAL OF VETERINARY INTERNAL MEDICINE

The effect of Tlr4 and/or C3 deficiency and of neonatal gene therapy on skeletal disease in mucopolysaccharidosis VII mice

(2015) Elizabeth M. Xing et al.   MOLECULAR GENETICS AND METABOLISM

Arylsulfatase A Overexpressing Human iPSC-derived Neural Cells Reduce CNS Sulfatide Storage in a Mouse Model of Metachromatic Leukodystrophy

(2015) Jonas Doerr et al.   MOLECULAR THERAPY

Elusive roles for reactive astrocytes in neurodegenerative diseases

(2015) Lucile Ben Haim et al.   Frontiers in Cellular Neuroscience

Astrocytes in Neurodegenerative Disease: Table 1.

(2015) Hemali Phatnani et al.   Cold Spring Harbor Perspectives in Biology

Neuroinflammatory paradigms in lysosomal storage diseases

(2015) Megan E. Bosch et al.   Frontiers in Neuroscience

Ex Vivo Gene Therapy Using Patient iPSC-Derived NSCs Reverses Pathology in the Brain of a Homologous Mouse Model

(2015) Tagan A. Griffin et al.   Stem Cell Reports

Activity and High-Order Effective Connectivity Alterations in Sanfilippo C Patient-Specific Neuronal Networks

(2015) Isaac Canals et al.   Stem Cell Reports

Stem Cells on the Brain: Modeling Neurodevelopmental and Neurodegenerative Diseases Using Human Induced Pluripotent Stem Cells

(2014) Priya Srikanth et al.   JOURNAL OF NEUROGENETICS

The generation of induced pluripotent stem cells (iPSCs) from patients with infantile and late-onset types of Pompe disease and the effects of treatment with acid-α-glucosidase in Pompe's iPSCs

(2014) Takashi Higuchi et al.   MOLECULAR GENETICS AND METABOLISM

A Modified Method for Implantation of Pluripotent Stem Cells Under the Rodent Kidney Capsule

(2014) Fuli Zhu et al.   STEM CELLS AND DEVELOPMENT

The Biogenesis of Lysosomes and Lysosome-Related Organelles

(2014) J. P. Luzio et al.   Cold Spring Harbor Perspectives in Biology

Genetic and Chemical Correction of Cholesterol Accumulation and Impaired Autophagy in Hepatic and Neural Cells Derived from Niemann-Pick Type C Patient-Specific iPS Cells

(2014) Dorothea Maetzel et al.   Stem Cell Reports

Proof of concept for AAV2/5-mediated gene therapy in iPSC-derived retinal pigment epithelium of a choroideremia patient

(2014) Nicolas Cereso et al.   Molecular Therapy-Methods & Clinical Development

Extracellular matrix components: An intricate network of possible biomarkers for lysosomal storage disorders?

(2013) Spyros P. Batzios et al.   FEBS LETTERS

Central Nervous System Delivery of Helper-Dependent Canine Adenovirus Corrects Neuropathology and Behavior in Mucopolysaccharidosis Type VII Mice

(2013) Lorena Ariza et al.   HUMAN GENE THERAPY

Disruption of the Coxsackievirus and Adenovirus Receptor-Homodimeric Interaction Triggers Lipid Microdomain- and Dynamin-dependent Endocytosis and Lysosomal Targeting

(2013) Sara Salinas et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Mucopolysaccharide diseases: A complex interplay between neuroinflammation, microglial activation and adaptive immunity

(2013) Louise D. Archer et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Autophagy in astrocytes

(2012) Chiara Di Malta et al.   Autophagy

Neuronopathic Gaucher's disease: induced pluripotent stem cells for disease modelling and testing chaperone activity of small compounds

(2012) Gustavo Tiscornia et al.   HUMAN MOLECULAR GENETICS

Generation and genetic modification of 3D cultures of human dopaminergic neurons derived from neural progenitor cells

(2012) Catarina Brito et al.   METHODS

Fiji: an open-source platform for biological-image analysis

(2012) Johannes Schindelin et al.   NATURE METHODS

Dysregulation of Gene Expression in a Lysosomal Storage Disease Varies between Brain Regions Implicating Unexpected Mechanisms of Neuropathology

(2012) Michael K. Parente et al.   PLoS One

Modeling neuronal defects associated with a lysosomal disorder using patient-derived induced pluripotent stem cells

(2011) Thomas Lemonnier et al.   HUMAN MOLECULAR GENETICS

Protein Transfer Into Human Cells by VSV-G-induced Nanovesicles

(2011) Philippe-Emmanuel Mangeot et al.   MOLECULAR THERAPY

Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)

(2010) J. Tolar et al.   BLOOD

A Call to Standardize Teratoma Assays Used to Define Human Pluripotent Cell Lines

(2010) Franz-Josef Müller et al.   Cell Stem Cell

Common and Uncommon Pathogenic Cascades in Lysosomal Storage Diseases

(2010) Einat B. Vitner et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Astrocytes: biology and pathology

(2009) Michael V. Sofroniew et al.   ACTA NEUROPATHOLOGICA

Mutations and polymorphisms in GUSB gene in mucopolysaccharidosis VII (Sly Syndrome)

(2009) Shunji Tomatsu et al.   HUMAN MUTATION

Mechanism of shortened bones in mucopolysaccharidosis VII

(2009) Jason A. Metcalf et al.   MOLECULAR GENETICS AND METABOLISM

Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling

(2009) Stuart M Chambers et al.   NATURE BIOTECHNOLOGY

Induced pluripotent stem cell technology for the study of human disease

(2009) Gabsang Lee et al.   NATURE METHODS

Generation of cortical neurons from mouse embryonic stem cells

(2009) Nicolas Gaspard et al.   Nature Protocols

Involvement of the Toll-like receptor 4 pathway and use of TNF-  antagonists for treatment of the mucopolysaccharidoses

(2009) C. M. Simonaro et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Gene Network Regulating Lysosomal Biogenesis and Function

(2009) Marco Sardiello et al.   SCIENCE

Lysosomal disorders: From storage to cellular damage

(2008) Andrea Ballabio et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Induction of pluripotent stem cells by defined factors is greatly improved by small-molecule compounds

(2008) Danwei Huangfu et al.   NATURE BIOTECHNOLOGY

Early Neurodegeneration Progresses Independently of Microglial Activation by Heparan Sulfate in the Brain of Mucopolysaccharidosis IIIB Mice

(2008) Jérôme Ausseil et al.   PLoS One

Teratoma Formation by Human Embryonic Stem Cells Is Site Dependent and Enhanced by the Presence of Matrigel

(2008) Tatyana A. Prokhorova et al.   STEM CELLS AND DEVELOPMENT