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

Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human SGSH and SUMF1 cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial

(2014) Marc Tardieu et al.   HUMAN GENE THERAPY

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

Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial

(2014) Stéphane Palfi et al.   LANCET

Extracellular Vesicles from Neural Stem Cells Transfer IFN-γ via Ifngr1 to Activate Stat1 Signaling in Target Cells

(2014) Chiara Cossetti et al.   MOLECULAR CELL

Early axonal loss accompanied by impaired endocytosis, abnormal axonal transport, and decreased microtubule stability occur in the model of Krabbe's disease

(2014) Carla Andreia Teixeira et al.   NEUROBIOLOGY OF DISEASE

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

(2013) F. Mingozzi et al.   BLOOD

Reversibility of neuropathology in Tay–Sachs-related diseases

(2013) María-Begoña Cachón-González et al.   HUMAN MOLECULAR GENETICS

Region- and Age-Dependent Alterations of Glial-Neuronal Metabolic Interactions Correlate with CNS Pathology in a Mouse Model of Globoid Cell Leukodystrophy

(2013) Tore Wergeland Meisingset et al.   JOURNAL OF CEREBRAL BLOOD FLOW AND METABOLISM

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

Multipotent Stromal Cells Alleviate Inflammation, Neuropathology, and Symptoms Associated with Globoid Cell Leukodystrophy in the Twitcher Mouse

(2013) Brittni A. Scruggs et al.   STEM CELLS

The galactocerebrosidase enzyme contributes to maintain a functional neurogenic niche during early post-natal CNS development

(2012) Sara Santambrogio et al.   HUMAN MOLECULAR GENETICS

Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy

(2012) Elizabeth Y. Qin et al.   MOLECULAR GENETICS AND METABOLISM

Extended Normal Life After AAVrh10-mediated Gene Therapy in the Mouse Model of Krabbe Disease

(2012) Mohammad A Rafi et al.   MOLECULAR THERAPY

Psychosine induces the dephosphorylation of neurofilaments by deregulation of PP1 and PP2A phosphatases

(2012) Ludovico Cantuti-Castelvetri et al.   NEUROBIOLOGY OF DISEASE

Microglial repopulation model reveals a robust homeostatic process for replacing CNS myeloid cells

(2012) N. H. Varvel et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2012) Martin McGlynn   Regenerative Medicine

Activating Transcription Factor 3 and the Nervous System

(2012) David Hunt et al.   Frontiers in Molecular Neuroscience

Bone Marrow Transplantation Alters the Tremor Phenotype in the Murine Model of Globoid-Cell Leukodystrophy

(2012) Adarsh S. Reddy et al.   Journal of Clinical Medicine

Axonopathy is a compounding factor in the pathogenesis of Krabbe disease

(2011) Ludovico Cantuti Castelvetri et al.   ACTA NEUROPATHOLOGICA

Gene therapy for leukodystrophies

(2011) A. Biffi et al.   HUMAN MOLECULAR GENETICS

Bone Marrow Transplantation Augments the Effect of Brain- and Spinal Cord-Directed Adeno-Associated Virus 2/5 Gene Therapy by Altering Inflammation in the Murine Model of Globoid-Cell Leukodystrophy

(2011) A. S. Reddy et al.   JOURNAL OF NEUROSCIENCE

A Signaling Cascade of Nuclear Calcium-CREB-ATF3 Activated by Synaptic NMDA Receptors Defines a Gene Repression Module That Protects against Extrasynaptic NMDA Receptor-Induced Neuronal Cell Death and Ischemic Brain Damage

(2011) S.-J. Zhang et al.   JOURNAL OF NEUROSCIENCE

CNS-targeted AAV5 gene transfer results in global dispersal of vector and prevention of morphological and function deterioration in CNS of globoid cell leukodystrophy mouse model

(2011) Dar-Shong Lin et al.   MOLECULAR GENETICS AND METABOLISM

Infiltrating monocytes trigger EAE progression, but do not contribute to the resident microglia pool

(2011) Bahareh Ajami et al.   NATURE NEUROSCIENCE

Systemic Delivery of Bone Marrow-Derived Mesenchymal Stromal Cells Diminishes Neuropathology in a Mouse Model of Krabbe's Disease

(2011) Catarina Oliveira Miranda et al.   STEM CELLS

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

(2011) Margherita Neri et al.   STEM CELLS

The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cell niche

(2010) I. Visigalli et al.   BLOOD

Activation transcription factor-3 activation and the development of spinal cord degeneration in a rat model of amyotrophic lateral sclerosis

(2010) A. Malaspina et al.   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

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

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

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

Monitoring disease evolution and treatment response in lysosomal disorders by the peripheral benzodiazepine receptor ligand PK11195

(2009) Ilaria Visigalli et al.   NEUROBIOLOGY OF DISEASE

Infiltrating Blood-Derived Macrophages Are Vital Cells Playing an Anti-inflammatory Role in Recovery from Spinal Cord Injury in Mice

(2009) Ravid Shechter et al.   PLOS MEDICINE

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