Blood-brain barrier delivery for lysosomal storage disorders with IgG-lysosomal enzyme fusion proteins
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Title
Blood-brain barrier delivery for lysosomal storage disorders with IgG-lysosomal enzyme fusion proteins
Authors
Keywords
Endothelium, Fusion proteins, Cerebrospinal fluid, Drug targeting, Receptor-mediated transport, Mathematical model
Journal
ADVANCED DRUG DELIVERY REVIEWS
Volume 184, Issue -, Pages 114234
Publisher
Elsevier BV
Online
2022-03-17
DOI
10.1016/j.addr.2022.114234
References
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Note: Only part of the references are listed.- Drug delivery for neuronopathic lysosomal storage diseases: evolving roles of the blood brain barrier and cerebrospinal fluid
- (2022) Yuji Sato et al. METABOLIC BRAIN DISEASE
- Cerebrospinal fluid outflow: a review of the historical and contemporary evidence for arachnoid villi, perineural routes, and dural lymphatics
- (2021) Steven T. Proulx CELLULAR AND MOLECULAR LIFE SCIENCES
- Clearance of heparan sulfate in the brain prevents neurodegeneration and neurocognitive impairment in MPS II mice
- (2021) Hideto Morimoto et al. MOLECULAR THERAPY
- Mucolipidosis Ⅱ and III with neurological symptoms due to spinal cord compression
- (2021) Sachiko Nakaoka et al. BRAIN & DEVELOPMENT
- Safety and efficacy of intrathecal idursulfase-IT in patients
- (2021) Joseph Muenzer et al. MOLECULAR GENETICS AND METABOLISM
- Updated results of Transpher A: multicenter, single-dose, phase 1/2 clinical trial of ABO-102 for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)
- (2021) Kevin Flanigan et al. MOLECULAR GENETICS AND METABOLISM
- Updated results of Transpher B: multicenter, single-dose, phase 1/2 clinical trial of ABO-101 for Sanfilippo syndrome type B (mucopolysaccharidosis IIIB)
- (2021) Maria de Castro et al. MOLECULAR GENETICS AND METABOLISM
- Iduronate-2-sulfatase fused with anti-hTfR antibody, pabinafusp alfa, for MPS-II: A phase 2 trial in Brazil
- (2021) Roberto Giugliani et al. MOLECULAR THERAPY
- Cell-Mediated Immunity to NAGLU Transgene Following Intracerebral Gene Therapy in Children With Mucopolysaccharidosis Type IIIB Syndrome
- (2021) Marie-Lise Gougeon et al. Frontiers in Immunology
- High dose genistein in Sanfilippo syndrome: A randomised controlled trial
- (2021) Arunabha Ghosh et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Can we learn lessons from the FDA’s approval of aducanumab?
- (2021) Kathy Y. Liu et al. Nature Reviews Neurology
- Impact of intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II
- (2021) Joo-Hyun Seo et al. Molecular Therapy-Methods & Clinical Development
- Mathematical Models of Blood-Brain Barrier Transport of Monoclonal Antibodies Targeting the Transferrin Receptor and the Insulin Receptor
- (2021) William M. Pardridge et al. Pharmaceuticals
- Kinetics of Blood–Brain Barrier Transport of Monoclonal Antibodies Targeting the Insulin Receptor and the Transferrin Receptor
- (2021) William M. Pardridge Pharmaceuticals
- Blood-Brain Barrier and Delivery of Protein and Gene Therapeutics to Brain
- (2020) William M. Pardridge Frontiers in Aging Neuroscience
- Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B
- (2020) Anita Grover et al. Drug Delivery and Translational Research
- Brain Pathology in Mucopolysaccharidoses (MPS) Patients with Neurological Forms
- (2020) Gustavo M. Viana et al. Journal of Clinical Medicine
- Molecular Bases of Neurodegeneration and Cognitive Decline, the Major Burden of Sanfilippo Disease
- (2020) Rachel Heon-Roberts et al. Journal of Clinical Medicine
- The relationships between neuroinflammation, beta-amyloid and tau deposition in Alzheimer’s disease: a longitudinal PET study
- (2020) Rola Ismail et al. Journal of Neuroinflammation
- Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice
- (2020) Julie C. Ullman et al. Science Translational Medicine
- The Isolated Brain Microvessel: A Versatile Experimental Model of the Blood-Brain Barrier
- (2020) William M. Pardridge Frontiers in Physiology
- Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial
- (2020) Christine í Dali et al. MOLECULAR GENETICS AND METABOLISM
- A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II
- (2020) Torayuki Okuyama et al. MOLECULAR THERAPY
- In vivo assessment of cerebrospinal fluid efflux to nasal mucosa in humans
- (2020) Erik Melin et al. Scientific Reports
- Stem Cell Therapy for Neurodegenerative Diseases: How Do Stem Cells Bypass the Blood-Brain Barrier and Home to the Brain?
- (2020) Yvonne Cashinn Chia et al. Stem Cells International
- Cerliponase alfa changes the natural history of children with neuronal ceroid lipofuscinosis type 2: The first French cohort
- (2020) Bastien Estublier et al. EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY
- Blood‐brain barrier transport using a high affinity, brain‐selective VNAR antibody targeting transferrin receptor 1
- (2020) Pawel Stocki et al. FASEB JOURNAL
- Can serial cerebral MRIs predict the neuronopathic phenotype of MPS II ?
- (2020) Audrey A. M. Vollebregt et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Temporospatial Development of Neuropathologic Findings in a Canine Model of Mucopolysaccharidosis IIIB
- (2020) Tyler A. Harm et al. VETERINARY PATHOLOGY
- Intravenous arylsulfatase A in metachromatic leukodystrophy: a phase 1/2 study
- (2020) Christine í Dali et al. Annals of Clinical and Translational Neurology
- Tetravalent Bispecific Tandem Antibodies Improve Brain Exposure and Efficacy in an Amyloid Transgenic Mouse Model
- (2020) Tuan-Minh Do et al. Molecular Therapy-Methods & Clinical Development
- Treatment of Alzheimer’s Disease and Blood–Brain Barrier Drug Delivery
- (2020) William M. Pardridge Pharmaceuticals
- Preclinical studies of a brain penetrating IgG Trojan horse-arylsulfatase fusion protein in the metachromatic leukodystrophy mouse
- (2019) Eric K.-W. Hui et al. MOLECULAR GENETICS AND METABOLISM
- Genotype‐phenotype relationships in mucopolysaccharidosis type I (MPS I): Insights from the International MPS I Registry
- (2019) Lorne A. Clarke et al. CLINICAL GENETICS
- The anatomy and immunology of vasculature in the central nervous system
- (2019) Panagiotis Mastorakos et al. Science Immunology
- Identification and evaluation of glutathione conjugate gamma-l-glutamyl-l-cysteine for improved drug delivery to the brain
- (2019) Saman Fatima et al. JOURNAL OF BIOMOLECULAR STRUCTURE & DYNAMICS
- The Effects of Leptin on Glial Cells in Neurological Diseases
- (2019) Yuki Fujita et al. Frontiers in Neuroscience
- Functional Expression of Choline Transporters in the Blood–Brain Barrier
- (2019) Masato Inazu Nutrients
- Pharmacokinetic Modeling of Intrathecally Administered Recombinant Human Arylsulfatase A (TAK‐611) in Children With Metachromatic Leukodystrophy
- (2019) Steven Troy et al. CLINICAL PHARMACOLOGY & THERAPEUTICS
- Solute Carrier Transporters as Potential Targets for the Treatment of Metabolic Disease
- (2019) Tina Schumann et al. PHARMACOLOGICAL REVIEWS
- Bi-functional IgG-lysosomal enzyme fusion proteins for brain drug delivery
- (2019) Ruben J. Boado et al. Scientific Reports
- Transferrin Receptors TfR1 and TfR2 Bind Transferrin through Differing Mechanisms
- (2018) Mark D. Kleven et al. BIOCHEMISTRY
- Plasma Pharmacokinetics of Valanafusp Alpha, a Human Insulin Receptor Antibody-Iduronidase Fusion Protein, in Patients with Mucopolysaccharidosis Type I
- (2018) William M. Pardridge et al. BIODRUGS
- Insulin receptor in the brain: Mechanisms of activation and the role in the CNS pathology and treatment
- (2018) Igor Pomytkin et al. CNS Neuroscience & Therapeutics
- Murine Sialidase Neu3 facilitates GM2 degradation and bypass in mouse model of Tay-Sachs disease
- (2018) Volkan Seyrantepe et al. EXPERIMENTAL NEUROLOGY
- Distinct progression patterns of brain disease in infantile and juvenile gangliosidoses: Volumetric quantitative MRI study
- (2018) Igor Nestrasil et al. MOLECULAR GENETICS AND METABOLISM
- A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II
- (2018) Hiroyuki Sonoda et al. MOLECULAR THERAPY
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- (2018) Dong Wang et al. NEURON
- Study of Intraventricular Cerliponase Alfa for CLN2 Disease
- (2018) Angela Schulz et al. NEW ENGLAND JOURNAL OF MEDICINE
- Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome
- (2018) Elsa G. Shapiro et al. Orphanet Journal of Rare Diseases
- Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial
- (2018) Roberto Giugliani et al. Orphanet Journal of Rare Diseases
- Nonclinical comparability studies of recombinant human arylsulfatase A addressing manufacturing process changes
- (2018) Teresa Wright et al. PLoS One
- The Impact of Carboxylesterases in Drug Metabolism and Pharmacokinetics
- (2018) Li Di CURRENT DRUG METABOLISM
- Evaluation of cerebrospinal fluid heparan sulfate as a biomarker of neuropathology in a murine model of mucopolysaccharidosis type II using high-sensitivity LC/MS/MS
- (2018) Noboru Tanaka et al. MOLECULAR GENETICS AND METABOLISM
- Anatomical changes and pathophysiology of the brain in mucopolysaccharidosis disorders
- (2018) Brian W. Bigger et al. MOLECULAR GENETICS AND METABOLISM
- Blood-Brain Barrier Transport, Plasma Pharmacokinetics, and Neuropathology Following Chronic Treatment of the Rhesus Monkey with a Brain Penetrating Humanized Monoclonal Antibody Against the Human Transferrin Receptor
- (2018) William M. Pardridge et al. MOLECULAR PHARMACEUTICS
- Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65
- (2018) Eloise Hudry et al. Molecular Therapy-Methods & Clinical Development
- Blood Vessels and Perivascular Phagocytes of Prefrontal White and Gray Matter in Suicide
- (2018) Tatiana P Schnieder et al. JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY
- Blood-brain barrier-associated pericytes internalize and clear aggregated amyloid-β42 by LRP1-dependent apolipoprotein E isoform-specific mechanism
- (2018) Qingyi Ma et al. Molecular Neurodegeneration
- Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial
- (2018) Torayuki Okuyama et al. MOLECULAR THERAPY
- Lysosomal storage diseases
- (2018) Frances M. Platt et al. Nature Reviews Disease Primers
- Use of LDL receptor–targeting peptide vectors for in vitro and in vivo cargo transport across the blood-brain barrier
- (2017) Yves Molino et al. FASEB JOURNAL
- Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System–Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer
- (2017) Kanut Laoharawee et al. HUMAN GENE THERAPY
- Astrocytic LRP1 Mediates Brain Aβ Clearance and Impacts Amyloid Deposition
- (2017) Chia-Chen Liu et al. JOURNAL OF NEUROSCIENCE
- Types A and B Niemann-Pick disease
- (2017) Edward H. Schuchman et al. MOLECULAR GENETICS AND METABOLISM
- Reduction in Brain Heparan Sulfate with Systemic Administration of an IgG Trojan Horse–Sulfamidase Fusion Protein in the Mucopolysaccharidosis Type IIIA Mouse
- (2017) Ruben J. Boado et al. MOLECULAR PHARMACEUTICS
- Brain and Organ Uptake in the Rhesus Monkey in Vivo of Recombinant Iduronidase Compared to an Insulin Receptor Antibody–Iduronidase Fusion Protein
- (2017) Ruben J. Boado et al. MOLECULAR PHARMACEUTICS
- Guanidinylated Neomycin Conjugation Enhances Intranasal Enzyme Replacement in the Brain
- (2017) Wenyong Tong et al. MOLECULAR THERAPY
- Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
- (2017) Jerry R. Mendell et al. NEW ENGLAND JOURNAL OF MEDICINE
- Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS)
- (2017) Joseph Muenzer et al. Orphanet Journal of Rare Diseases
- Management Strategies for CLN2 Disease
- (2017) Ruth E. Williams et al. PEDIATRIC NEUROLOGY
- FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides
- (2017) Annemieke Aartsma-Rus Nucleic Acid Therapeutics
- Development of the First World Health Organization Lentiviral Vector Standard: Toward the Production Control and Standardization of Lentivirus-Based Gene Therapy Products
- (2017) Yuan Zhao et al. Human Gene Therapy Methods
- The SLC28 (CNT) and SLC29 (ENT) nucleoside transporter families: a 30-year collaborative odyssey
- (2016) J. D. Young BIOCHEMICAL SOCIETY TRANSACTIONS
- CSF, blood-brain barrier, and brain drug delivery
- (2016) William M. Pardridge Expert Opinion on Drug Delivery
- Preclinical Testing of the Safety and Tolerability of Lentiviral Vector–Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies
- (2016) Ilaria Visigalli et al. HUMAN GENE THERAPY
- Asfotase Alfa Treatment Improves Survival for Perinatal and Infantile Hypophosphatasia
- (2016) Michael P. Whyte et al. JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM
- Structure of Human Acid Sphingomyelinase Reveals the Role of the Saposin Domain in Activating Substrate Hydrolysis
- (2016) Zi-Jian Xiong et al. JOURNAL OF MOLECULAR BIOLOGY
- Granular Layer Neurons Control Cerebellar Neurovascular Coupling Through an NMDA Receptor/NO-Dependent System
- (2016) Lisa Mapelli et al. JOURNAL OF NEUROSCIENCE
- Histochemical localization of palmitoyl protein thioesterase-1 activity
- (2016) Joshua T. Dearborn et al. MOLECULAR GENETICS AND METABOLISM
- Enzyme replacement for GM1-gangliosidosis: Uptake, lysosomal activation, and cellular disease correction using a novel β-galactosidase:RTB lectin fusion
- (2016) Jose Condori et al. MOLECULAR GENETICS AND METABOLISM
- Effect of anti-laronidase antibodies on efficacy and safety of laronidase enzyme replacement therapy for MPS I: A comprehensive meta-analysis of pooled data from multiple studies
- (2016) Yong Xue et al. MOLECULAR GENETICS AND METABOLISM
- A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA
- (2016) Simon A. Jones et al. MOLECULAR GENETICS AND METABOLISM
- Very High Plasma Concentrations of a Monoclonal Antibody against the Human Insulin Receptor Are Produced by Subcutaneous Injection in the Rhesus Monkey
- (2016) Ruben J. Boado et al. MOLECULAR PHARMACEUTICS
- Insulin Receptor Antibody−α-N-Acetylglucosaminidase Fusion Protein Penetrates the Primate Blood–Brain Barrier and Reduces Glycosoaminoglycans in Sanfilippo Type B Fibroblasts
- (2016) Ruben J. Boado et al. MOLECULAR PHARMACEUTICS
- The antibody aducanumab reduces Aβ plaques in Alzheimer’s disease
- (2016) Jeff Sevigny et al. NATURE
- Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy
- (2016) Claudia A. Chiriboga et al. NEUROLOGY
- Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo
- (2016) Michael B Tropak et al. Molecular Therapy-Methods & Clinical Development
- Butanolysis Derivatization: Improved Sensitivity in LC-MS/MS Quantitation of Heparan Sulfate in Urine from Mucopolysaccharidosis Patients
- (2015) Paul J. Trim et al. ANALYTICAL CHEMISTRY
- Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study
- (2015) M. Aldenhoven et al. BLOOD
- In vitro discrimination of the role of LRP1 at the BBB cellular level: Focus on brain capillary endothelial cells and brain pericytes
- (2015) Pietra Candela et al. BRAIN RESEARCH
- Blood–brain barrier endogenous transporters as therapeutic targets: a new model for small molecule CNS drug discovery
- (2015) William M Pardridge EXPERT OPINION ON THERAPEUTIC TARGETS
- A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II
- (2015) Joseph Muenzer et al. GENETICS IN MEDICINE
- Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan
- (2015) Cara M. Weismann et al. HUMAN MOLECULAR GENETICS
- Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy
- (2015) Randy J. Chandler et al. JOURNAL OF CLINICAL INVESTIGATION
- Probing the extracellular diffusion of antibodies in brain using in vivo integrative optical imaging and ex vivo fluorescence imaging
- (2015) Daniel J. Wolak et al. JOURNAL OF CONTROLLED RELEASE
- Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis
- (2015) Jui-Yun Lu et al. MOLECULAR GENETICS AND METABOLISM
- Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis
- (2015) Brian R. Vuillemenot et al. MOLECULAR GENETICS AND METABOLISM
- Determination of arylsulfatase A pseudodeficiency allele and haplotype frequency in the Tunisian population
- (2015) Nizar Ben Halim et al. NEUROLOGICAL SCIENCES
- Functional identification of SLC43A3 as an equilibrative nucleobase transporter involved in purine salvage in mammals
- (2015) Junji Furukawa et al. Scientific Reports
- New aspects in fenestrated capillary and tissue dynamics in the sensory circumventricular organs of adult brains
- (2015) Seiji Miyata Frontiers in Neuroscience
- A Multiplex Assay for the Diagnosis of Mucopolysaccharidoses and Mucolipidoses
- (2015) Eveline J. Langereis et al. PLoS One
- Insulin receptor antibody-iduronate 2-sulfatase fusion protein: Pharmacokinetics, anti-drug antibody, and safety pharmacology in Rhesus monkeys
- (2014) Ruben J. Boado et al. BIOTECHNOLOGY AND BIOENGINEERING
- Comparison of Five Peptide Vectors for Improved Brain Delivery of the Lysosomal Enzyme Arylsulfatase A
- (2014) A. Bockenhoff et al. JOURNAL OF NEUROSCIENCE
- Insulin Receptor Antibody–Sulfamidase Fusion Protein Penetrates the Primate Blood–Brain Barrier and Reduces Glycosoaminoglycans in Sanfilippo Type A Cells
- (2014) Ruben J. Boado et al. MOLECULAR PHARMACEUTICS
- Increased Brain Penetration and Potency of a Therapeutic Antibody Using a Monovalent Molecular Shuttle
- (2014) Jens Niewoehner et al. NEURON
- A Monoclonal Antibody TrkB Receptor Agonist as a Potential Therapeutic for Huntington’s Disease
- (2014) Daniel Todd et al. PLoS One
- Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB
- (2014) S.-h. Kan et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Therapeutic bispecific antibodies cross the blood-brain barrier in nonhuman primates
- (2014) Y. J. Yu et al. Science Translational Medicine
- Recombinant human tripeptidyl peptidase-1 infusion to the monkey CNS: Safety, pharmacokinetics, and distribution
- (2014) Brian R. Vuillemenot et al. TOXICOLOGY AND APPLIED PHARMACOLOGY
- Genistein Induces Deleterious Effects during Its Acute Exposure in Swiss Mice
- (2014) Prabhat Singh et al. Biomed Research International
- Structure of sulfamidase provides insight into the molecular pathology of mucopolysaccharidosis IIIA
- (2014) Navdeep S. Sidhu et al. ACTA CRYSTALLOGRAPHICA SECTION D-BIOLOGICAL CRYSTALLOGRAPHY
- A review of β-amyloid neuroimaging in Alzheimer's disease
- (2014) Paul A. Adlard et al. Frontiers in Neuroscience
- IgG Subclasses and Allotypes: From Structure to Effector Functions
- (2014) Gestur Vidarsson et al. Frontiers in Immunology
- Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating Mucopolysaccharidosis IIIB: Toxicology, Biodistribution, and Immunological Assessments in Primates
- (2014) Darren A. Murrey et al. Human Gene Therapy Clinical Development
- 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
- Glycan Structure Determinants for Cation-Independent Mannose 6-Phosphate Receptor Binding and Cellular Uptake of a Recombinant Protein
- (2013) Qun Zhou et al. BIOCONJUGATE CHEMISTRY
- Effects of baclofen on mechanical noxious and innocuous transmission in the spinal dorsal horn of the adult rat:in vivopatch-clamp analysis
- (2013) Kaori Fukuhara et al. EUROPEAN JOURNAL OF NEUROSCIENCE
- Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
- (2013) S J Gray et al. GENE THERAPY
- Spinal muscular atrophy: a motor neuron disorder or a multi-organ disease?
- (2013) Monir Shababi et al. JOURNAL OF ANATOMY
- Atorvastatin and pitavastatin reduce oxidative stress and improve IR/LDL-R signals in Alzheimer’s disease
- (2013) Tomoko Kurata et al. NEUROLOGICAL RESEARCH
- Addressing Safety Liabilities of TfR Bispecific Antibodies That Cross the Blood-Brain Barrier
- (2013) J. A. Couch et al. Science Translational Medicine
- Decreasing Activity and Altered Protein Processing of Human Iduronate-2-sulfatase Mutations Demonstrated by Expression in COS7 Cells
- (2012) Ratana Charoenwattanasatien et al. BIOCHEMICAL GENETICS
- IgG-Enzyme Fusion Protein: Pharmacokinetics and Anti-Drug Antibody Response in Rhesus Monkeys
- (2012) Ruben J. Boado et al. BIOCONJUGATE CHEMISTRY
- Pharmacokinetics and brain uptake in the rhesus monkey of a fusion protein of arylsulfatase a and a monoclonal antibody against the human insulin receptor
- (2012) Ruben J. Boado et al. BIOTECHNOLOGY AND BIOENGINEERING
- Drug Transport across the Blood–Brain Barrier
- (2012) William M Pardridge JOURNAL OF CEREBRAL BLOOD FLOW AND METABOLISM
- Selective plasma pharmacokinetics and brain uptake in the mouse of enzyme fusion proteins derived from species-specific receptor-targeted antibodies
- (2012) Qing-Hui Zhou et al. JOURNAL OF DRUG TARGETING
- Plasma and urinary levels of dermatan sulfate and heparan sulfate derived disaccharides after long-term enzyme replacement therapy (ERT) in MPS I: correlation with the timing of ERT and with total urinary excretion of glycosaminoglycans
- (2012) Minke H. de Ru et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Biophysical and Ultrastructural Characterization of Adeno-Associated Virus Capsid Uncoating and Genome Release
- (2012) E. D. Horowitz et al. JOURNAL OF VIROLOGY
- Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl–protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis
- (2012) Jie Hu et al. MOLECULAR GENETICS AND METABOLISM
- Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A
- (2012) Jie Bu et al. MOLECULAR THERAPY
- Intrathecal recombinant human 4-sulfatase reduces accumulation of glycosaminoglycans in dura of mucopolysaccharidosis VI cats
- (2012) Dyane Auclair et al. PEDIATRIC RESEARCH
- Systemic Administration of Tripeptidyl Peptidase I in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis: Effect of Glycan Modification
- (2012) Yu Meng et al. PLoS One
- Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders
- (2012) Silvia Muro Drug Delivery and Translational Research
- Current advances in using neurotrophic factors to treat neurodegenerative disorders
- (2012) April M Weissmiller et al. Translational Neurodegeneration
- Genistein in Sanfilippo disease: A randomized controlled crossover trial
- (2011) Jessica de Ruijter et al. ANNALS OF NEUROLOGY
- Expression in CHO cells and pharmacokinetics and brain uptake in the Rhesus monkey of an IgG-iduronate-2-sulfatase fusion protein
- (2011) Jeff Zhiqiang Lu et al. BIOTECHNOLOGY AND BIOENGINEERING
- Analysis of Glycosaminoglycans in Cerebrospinal Fluid from Patients with Mucopolysaccharidoses by Isotope-Dilution Ultra-Performance Liquid Chromatography-Tandem Mass Spectrometry
- (2011) H. Zhang et al. CLINICAL CHEMISTRY
- Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusion
- (2011) Robin J. Ziegler et al. EXPERIMENTAL NEUROLOGY
- Impact of high-dose, chemically modified sulfamidase on pathology in a murine model of MPS IIIA
- (2011) Tina Rozaklis et al. EXPERIMENTAL NEUROLOGY
- Childhood leukodystrophies: a clinical perspective
- (2011) Alfried Kohlschütter et al. Expert Review of Neurotherapeutics
- Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates
- (2011) Lluis Samaranch et al. HUMAN GENE THERAPY
- The collagenic architecture of human dura mater
- (2011) Marina Protasoni et al. JOURNAL OF NEUROSURGERY
- Evaluation of Miglustat Treatment in Patients with Type III Mucopolysaccharidosis: A Randomized, Double-Blind, Placebo-Controlled Study
- (2011) Nathalie Guffon et al. JOURNAL OF PEDIATRICS
- Reversal of Lysosomal Storage in Brain of Adult MPS-I Mice with Intravenous Trojan Horse-Iduronidase Fusion Protein
- (2011) Ruben J. Boado et al. MOLECULAR PHARMACEUTICS
- How the binding of human transferrin primes the transferrin receptor potentiating iron release at endosomal pH
- (2011) Brian E. Eckenroth et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Boosting Brain Uptake of a Therapeutic Antibody by Reducing Its Affinity for a Transcytosis Target
- (2011) Y. J. Yu et al. Science Translational Medicine
- Heparan Sulfate Proteoglycans
- (2011) S. Sarrazin et al. Cold Spring Harbor Perspectives in Biology
- Genistein reduces glycosaminoglycan levels in a mouse model of mucopolysaccharidosis type II
- (2010) A Friso et al. BRITISH JOURNAL OF PHARMACOLOGY
- 4-methylumbelliferyl α-N-acetylglucosaminidase activity for diagnosis of Sanfilippo B disease
- (2010) Jane Marsh et al. CLINICAL GENETICS
- Secondary Storage of Dermatan Sulfate in Sanfilippo Disease
- (2010) William C. Lamanna et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Insight into the patterns of cerebrospinal fluid flow in the human ventricular system using MR velocity mapping
- (2010) Andreas Stadlbauer et al. NEUROIMAGE
- Intracisternal enzyme replacement therapy in lysosomal storage diseases: routes of absorption into brain
- (2010) R. D. Jolly et al. NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY
- Metachromatic Leukodystrophy – An Update
- (2010) V. Gieselmann et al. NEUROPEDIATRICS
- Genistein Improves Neuropathology and Corrects Behaviour in a Mouse Model of Neurodegenerative Metabolic Disease
- (2010) Marcelina Malinowska et al. PLoS One
- The blood-brain barrier and glutamate
- (2009) Richard A Hawkins AMERICAN JOURNAL OF CLINICAL NUTRITION
- Genetic Engineering of a Bifunctional IgG Fusion Protein with Iduronate-2-Sulfatase
- (2009) Jeff Zhiqiang Lu et al. BIOCONJUGATE CHEMISTRY
- Towards understanding the neuronal ceroid lipofuscinoses
- (2009) Alfried Kohlschütter et al. BRAIN & DEVELOPMENT
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- (2008) J. H. Grubb et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
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