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

Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

(2016) Maria Sessa et al.   LANCET

Adult Mammalian Neural Stem Cells and Neurogenesis: Five Decades Later

(2015) Allison M. Bond et al.   Cell Stem Cell

Pluripotent stem cell-derived radial glia-like cells as stable intermediate for efficient generation of human oligodendrocytes

(2015) Raphaela Gorris et al.   GLIA

Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy

(2015) A. Ricca et al.   HUMAN MOLECULAR GENETICS

Human neural stem cell transplantation in ALS: initial results from a phase I trial

(2015) Letizia Mazzini et al.   Journal of Translational Medicine

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

Generation and isolation of oligodendrocyte progenitor cells from human pluripotent stem cells

(2015) Panagiotis Douvaras et al.   Nature Protocols

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

Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy

(2014) A. Lattanzi et al.   HUMAN MOLECULAR GENETICS

Neural progenitor cells from human induced pluripotent stem cells generated less autogenous immune response

(2014) Ke Huang et al.   Science China-Life Sciences

Origin-Dependent Neural Cell Identities in Differentiated Human iPSCs In Vitro and after Transplantation into the Mouse Brain

(2014) Gunnar Hargus et al.   Cell Reports

Induced Neural Stem Cells Achieve Long-Term Survival and Functional Integration in the Adult Mouse Brain

(2014) Kathrin Hemmer et al.   Stem Cell Reports

Neurodevelopmental Outcomes of Umbilical Cord Blood Transplantation in Metachromatic Leukodystrophy

(2013) Holly R. Martin et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Human iPSC-Derived Oligodendrocyte Progenitor Cells Can Myelinate and Rescue a Mouse Model of Congenital Hypomyelination

(2013) Su Wang et al.   Cell Stem Cell

Neuro-immune interactions of neural stem cell transplants: From animal disease models to human trials

(2013) Elena Giusto et al.   EXPERIMENTAL NEUROLOGY

How stem cells speak with host immune cells in inflammatory brain diseases

(2013) Stefano Pluchino et al.   GLIA

Central nervous system stem cell transplantation for children with neuronal ceroid lipofuscinosis

(2013) Nathan R. Selden et al.   Journal of Neurosurgery-Pediatrics

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Clinical translation of human neural stem cells

(2013) Ann Tsukamoto et al.   Stem Cell Research & Therapy

Induced Pluripotent Stem Cell-Derived Neural Cells Survive and Mature in the Nonhuman Primate Brain

(2013) Marina E. Emborg et al.   Cell Reports

Juvenile metachromatic leukodystrophy 10 years post transplant compared with a non-transplanted cohort

(2012) I Krägeloh-Mann et al.   BONE MARROW TRANSPLANTATION

Neural stem cell transplantation in central nervous system disorders

(2012) Donatella De Feo et al.   CURRENT OPINION IN NEUROLOGY

Cross-talk between neural stem cells and immune cells: the key to better brain repair?

(2012) Zaal Kokaia et al.   NATURE NEUROSCIENCE

Capture of Neuroepithelial-Like Stem Cells from Pluripotent Stem Cells Provides a Versatile System for In Vitro Production of Human Neurons

(2012) Anna Falk et al.   PLoS One

StemCells, Inc.: clinical trials of stem cell therapies for CNS disorders

(2012) Martin McGlynn   Regenerative Medicine

Human Neural Stem Cells Induce Functional Myelination in Mice with Severe Dysmyelination

(2012) N. Uchida et al.   Science Translational Medicine

Extracellular Membrane Vesicles and Immune Regulation in the Brain

(2012) Chiara Cossetti et al.   Frontiers in Physiology

Genomic instability in induced stem cells

(2011) C E Pasi et al.   CELL DEATH AND DIFFERENTIATION

Gene therapy for lysosomal storage disorders

(2011) Angela Gritti   EXPERT OPINION ON BIOLOGICAL THERAPY

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

(2011) Thomas Lemonnier et al.   HUMAN MOLECULAR GENETICS

Brain Regeneration in Physiology and Pathology: The Immune Signature Driving Therapeutic Plasticity of Neural Stem Cells

(2011) Gianvito Martino et al.   PHYSIOLOGICAL REVIEWS

Transgene Excision Has No Impact on In Vivo Integration of Human iPS Derived Neural Precursors

(2011) Tamara Major et al.   PLoS One

Neural Stem Cell Gene Therapy Ameliorates Pathology and Function in a Mouse Model of Globoid Cell Leukodystrophy

(2011) Margherita Neri et al.   STEM CELLS

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

(2010) J. Tolar et al.   BLOOD

iPSC lines that do not silence the expression of the ectopic reprogramming factors may display enhanced propensity to genomic instability

(2010) Veronica Ramos-Mejia et al.   CELL RESEARCH

Long-term culture and differentiation of CNS precursors derived from anterior human neural rosettes following exposure to ventralizing factors

(2010) Silvia Colleoni et al.   EXPERIMENTAL CELL RESEARCH

Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models

(2010) Annalisa Lattanzi et al.   HUMAN MOLECULAR GENETICS

Molecular and structural analysis of metachromatic leukodystrophy patients in Indian population

(2010) Pallavi Shukla et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

Erratum: Neural stem cell systems: physiological players or in vitro entities?

(2010) Luciano Conti et al.   NATURE REVIEWS NEUROSCIENCE

Robust Generation of Oligodendrocyte Progenitors from Human Neural Stem Cells and Engraftment in Experimental Demyelination Models in Mice

(2010) Margherita Neri et al.   PLoS One

Identification of Hematopoietic Stem Cell-Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy

(2010) B. Gentner et al.   Science Translational Medicine

Human neural stem cells ameliorate autoimmune encephalomyelitis in non-human primates

(2009) Stefano Pluchino et al.   ANNALS OF NEUROLOGY

Delayed post-ischaemic neuroprotection following systemic neural stem cell transplantation involves multiple mechanisms

(2009) Marco Bacigaluppi et al.   BRAIN

Neuroprotection of Host Cells by Human Central Nervous System Stem Cells in a Mouse Model of Infantile Neuronal Ceroid Lipofuscinosis

(2009) Stanley J. Tamaki et al.   Cell Stem Cell

Genome modification in human embryonic stem cells

(2009) Toyoaki Tenzen et al.   JOURNAL OF CELLULAR PHYSIOLOGY

A rosette-type, self-renewing human ES cell-derived neural stem cell with potential for in vitro instruction and synaptic integration

(2009) P. Koch et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Neural Stem Cell Transplantation Benefits a Monogenic Neurometabolic Disorder During the Symptomatic Phase of Disease

(2009) Mylvaganam Jeyakumar et al.   STEM CELLS

CNS-directed gene therapy for lysosomal storage diseases

(2008) Mark S Sands et al.   ACTA PAEDIATRICA

Sorting of lysosomal proteins

(2008) Thomas Braulke et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Multipotential Neural Precursors Transplanted into the Metachromatic Leukodystrophy Brain Fail to Generate Oligodendrocytes but Contribute to Limit Brain Dysfunction

(2008) M.I. Givogri et al.   DEVELOPMENTAL NEUROSCIENCE

Human ES cell-derived neural rosettes reveal a functionally distinct early neural stem cell stage

(2008) Y. Elkabetz et al.   GENES & DEVELOPMENT

Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA

(2008) Stefan Worgall et al.   HUMAN GENE THERAPY