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

Axonopathy is a compounding factor in the pathogenesis of Krabbe disease

(2011) Ludovico Cantuti Castelvetri et al.   ACTA NEUROPATHOLOGICA

ReNeuron and StemCells get green light for neural stem cell trials

(2011) George S Mack   NATURE BIOTECHNOLOGY

Ex vivo gene transfer and correction for cell-based therapies

(2011) Luigi Naldini   NATURE REVIEWS GENETICS

Mesenchymal stem cells for the sustained in vivo delivery of bioactive factors

(2010) Todd Meyerrose et al.   ADVANCED DRUG DELIVERY REVIEWS

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model

(2010) I. Visigalli et al.   BLOOD

Mesenchymal stem cells at the intersection of cell and gene therapy

(2010) Timothy J Myers et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

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

Stem cells in human neurodegenerative disorders — time for clinical translation?

(2010) Olle Lindvall et al.   JOURNAL OF CLINICAL INVESTIGATION

Persistence of psychosine in brain lipid rafts is a limiting factor in the therapeutic recovery of a mouse model for Krabbe disease

(2010) A.B. White et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Human neural stem cells genetically modified to overexpress brain-derived neurotrophic factor promote functional recovery and neuroprotection in a mouse stroke model

(2010) Hong J. Lee et al.   JOURNAL OF NEUROSCIENCE RESEARCH

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

Long-Term Survival of Human Neural Stem Cells in the Ischemic Rat Brain upon Transient Immunosuppression

(2010) Laura Rota Nodari et al.   PLoS One

Communication via gap junctions underlies early functional and beneficial interactions between grafted neural stem cells and the host

(2010) J. Jaderstad et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

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

Mesenchymal Lineage Stem Cells Have Pronounced Anti-Inflammatory Effects in the Twitcher Mouse Model of Krabbe's Disease

(2010) Cynthia B. Ripoll et al.   STEM CELLS

Diffusion Tensor Imaging Detects Abnormalities in the Corticospinal Tracts of Neonates with Infantile Krabbe Disease

(2009) M.L. Escolar et al.   AMERICAN JOURNAL OF NEURORADIOLOGY

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

Effects of treatments on inflammatory and apoptotic markers in the CNS of mice with globoid cell leukodystrophy

(2009) Paola Luzi et al.   BRAIN RESEARCH

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

Specific Determination of  -Galactocerebrosidase Activity via Competitive Inhibition of  -Galactosidase

(2009) S. Martino et al.   CLINICAL CHEMISTRY

The long-term outcomes of presymptomatic infants transplanted for Krabbe disease: Report of the workshop held on July 11 and 12, 2008, Holiday Valley, New York

(2009) Patricia K Duffner et al.   GENETICS IN MEDICINE

Effects of developmental age, brain region, and time in culture on long-term proliferation and multipotency of neural stem cell populations

(2009) Angela Gritti et al.   JOURNAL OF COMPARATIVE NEUROLOGY

Neural precursor cell cultures from GM2 gangliosidosis animal models recapitulate the biochemical and molecular hallmarks of the brain pathology

(2009) Sabata Martino et al.   JOURNAL OF NEUROCHEMISTRY

Combined hematopoietic and lentiviral gene-transfer therapies in newborn Twitcher mice reveal contemporaneous neurodegeneration and demyelination in Krabbe disease

(2009) F. Galbiati et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Significant correction of pathology in brains of twitcher mice following injection of genetically modified mouse neural progenitor cells

(2009) Michelina Strazza et al.   MOLECULAR GENETICS AND METABOLISM

Leukodystrophies

(2009) Daniel J. Costello et al.   NEUROLOGIST

Immune Regulatory Neural Stem/Precursor Cells Protect from Central Nervous System Autoimmunity by Restraining Dendritic Cell Function

(2009) Stefano Pluchino et al.   PLoS One

Transplant Outcomes in Leukodystrophies

(2009) Paul J. Orchard et al.   SEMINARS IN HEMATOLOGY

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

Neonatal Chimerization with Human Glial Progenitor Cells Can Both Remyelinate and Rescue the Otherwise Lethally Hypomyelinated Shiverer Mouse

(2008) Martha S. Windrem et al.   Cell Stem Cell

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

Efficient In Vitro Labeling of Human Neural Precursor Cells with Superparamagnetic Iron Oxide Particles: Relevance for In Vivo Cell Tracking

(2007) Margherita Neri et al.   STEM CELLS