Murine neural stem cells model Hunter disease in vitro: glial cell-mediated neurodegeneration as a possible mechanism involved

Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases

(2012) Rosella Tomanin et al.   ACTA PAEDIATRICA

Enzyme replacement therapy (ERT) procedure for mucopolysaccharidosis type II (MPS II) by intraventricular administration (IVA) in murine MPS II

(2012) Takashi Higuchi et al.   MOLECULAR GENETICS AND METABOLISM

The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensus

(2011) Joseph Muenzer et al.   EUROPEAN JOURNAL OF PEDIATRICS

Idursulfase treatment of Hunter syndrome in children younger than 6 years: Results from the Hunter Outcome Survey

(2011) Joseph Muenzer et al.   GENETICS IN MEDICINE

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

(2011) Thomas Lemonnier et al.   HUMAN MOLECULAR GENETICS

PDGFR  Expression Distinguishes GFAP-Expressing Neural Stem Cells from PDGF-Responsive Neural Precursors in the Adult Periventricular Area

(2011) A. Chojnacki et al.   JOURNAL OF NEUROSCIENCE

Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease

(2011) Maurizio Scarpa et al.   Orphanet Journal of Rare Diseases

Overview of the mucopolysaccharidoses

(2011) J. Muenzer   RHEUMATOLOGY

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

(2010) Joseph Muenzer et al.   GENETICS IN MEDICINE

Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy

(2010) Vinicia Assunta Polito et al.   HUMAN MOLECULAR GENETICS

Common and Uncommon Pathogenic Cascades in Lysosomal Storage Diseases

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

Quiescence and Activation of Stem and Precursor Cell Populations in the Subependymal Zone of the Mammalian Brain Are Associated with Distinct Cellular and Extracellular Matrix Signals

(2010) I. Kazanis et al.   JOURNAL OF NEUROSCIENCE

Regulation of Mammalian Autophagy in Physiology and Pathophysiology

(2010) Brinda Ravikumar et al.   PHYSIOLOGICAL REVIEWS

Induced pluripotent stem cells derived from mouse models of lysosomal storage disorders

(2010) Xing-Li Meng et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

IDS Crossing of the Blood-Brain Barrier Corrects CNS Defects in MPSII Mice

(2009) Vinicia Assunta Polito et al.   AMERICAN JOURNAL OF HUMAN GENETICS

DNA-damage response, survival and differentiation in vitro of a human neural stem cell line in relation to ATM expression

(2009) L Carlessi et al.   CELL DEATH AND DIFFERENTIATION

Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type II

(2008) J Edmond Wraith   ACTA PAEDIATRICA

Lysosomal disorders: From storage to cellular damage

(2008) Andrea Ballabio 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