Neural cells generated from human induced pluripotent stem cells as a model of CNS involvement in mucopolysaccharidosis type II

An isogenic blood-brain barrier model comprising brain endothelial cells, astrocytes, and neurons derived from human induced pluripotent stem cells

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Glycosaminoglycans and mucopolysaccharidosis type III

(2016) Grzegorz Wegrzyn   Frontiers in Bioscience-Landmark

Modeling autosomal dominant optic atrophy using induced pluripotent stem cells and identifying potential therapeutic targets

(2016) Jing Chen et al.   Stem Cell Research & Therapy

Early Intervention for Spinal Cord Injury with Human Induced Pluripotent Stem Cells Oligodendrocyte Progenitors

(2015) Angelo H. All et al.   PLoS One

Current and potential therapeutic strategies for mucopolysaccharidoses

(2014) H. Noh et al.   JOURNAL OF CLINICAL PHARMACY AND THERAPEUTICS

Effective clearance of GL-3 in a human iPSC-derived cardiomyocyte model of Fabry disease

(2014) Jean-Michel Itier et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Blood-Brain Barrier Molecular Trojan Horse Enables Imaging of Brain Uptake of Radioiodinated Recombinant Protein in the Rhesus Monkey

(2013) Ruben J. Boado et al.   BIOCONJUGATE CHEMISTRY

The effect of idursulfase on growth in patients with Hunter syndrome: Data from the Hunter Outcome Survey (HOS)

(2013) Simon A. Jones et al.   MOLECULAR GENETICS AND METABOLISM

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

(2013) E Fusar Poli et al.   Cell Death & Disease

Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: A nationwide survey in Japan

(2012) Akemi Tanaka et al.   MOLECULAR GENETICS AND METABOLISM

Efficient derivation of NPCs, spinal motor neurons and midbrain dopaminergic neurons from hESCs at 3% oxygen

(2011) S R L Stacpoole et al.   Nature Protocols

Heparan Sulfate Proteoglycans

(2011) S. Sarrazin et al.   Cold Spring Harbor Perspectives in Biology

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

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

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

Analysis of Pluripotent Stem Cells by using Cryosections of Embryoid Bodies

(2010) Ismael C. Gomes et al.   Jove-Journal of Visualized Experiments

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

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

Japan Elaprase® Treatment (JET) study: Idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II)

(2009) Torayuki Okuyama et al.   MOLECULAR GENETICS AND METABOLISM