Targeted enzyme delivery systems in lysosomal disorders: an innovative form of therapy for mucopolysaccharidosis

Enzyme-conjugated gold nanoparticles for combined enzyme and photothermal therapy of colon cancer cells

(2019) Mostafa Akbarzadeh Khiavi et al.   COLLOIDS AND SURFACES A-PHYSICOCHEMICAL AND ENGINEERING ASPECTS

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

Optimization of alginate microcapsules containing cells overexpressing α- l -iduronidase using Box-Behnken design

(2018) Dirnete Diel et al.   EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

The efficacy of intracerebroventricular idursulfase-beta enzyme replacement therapy in mucopolysaccharidosis II murine model: heparan sulfate in cerebrospinal fluid as a clinical biomarker of neuropathology

(2018) Young Bae Sohn et al.   JOURNAL OF INHERITED METABOLIC DISEASE

A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II

(2018) Hiroyuki Sonoda et al.   MOLECULAR THERAPY

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

Intrathecal/Intracerebroventricular enzyme replacement therapy for the mucopolysaccharidoses: efficacy, safety, and prospects

(2018) Roberto Giugliani et al.   Expert Opinion on Orphan Drugs

A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I

(2018) Steven Q. Le et al.   Molecular Therapy-Methods & Clinical Development

Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Mucopolysaccharidoses

(2018) Valeria De Pasquale et al.   Molecular Therapy-Methods & Clinical Development

Monoolein-based nanoparticles for drug delivery to the central nervous system: A platform for lysosomal storage disorder treatment

(2018) Bruna Donida et al.   EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS

Prodrug approach: An overview of recent cases

(2017) Valentina Abet et al.   EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Phase I and II clinical trials for the mucopolysaccharidoses

(2017) Fabiano Poswar et al.   EXPERT OPINION ON INVESTIGATIONAL DRUGS

Overview of immune abnormalities in lysosomal storage disorders

(2017) Donato Rigante et al.   IMMUNOLOGY LETTERS

Non-invasive approaches for drug delivery to the brain based on the receptor mediated transport

(2017) Fei Fang et al.   Materials Science & Engineering C-Materials for Biological Applications

Treatment of brain disease in the mucopolysaccharidoses

(2017) Maurizio Scarpa et al.   MOLECULAR GENETICS AND METABOLISM

A novel, randomized, placebo-controlled, blind-start, single-crossover phase 3 study to assess the efficacy and safety of UX003 (rhGUS) enzyme replacement therapy in patients with MPS VII

(2017) Paul Harmatz et al.   MOLECULAR GENETICS AND METABOLISM

Developmental and behavioral aspects of mucopolysaccharidoses with brain manifestations — Neurological signs and symptoms

(2017) Elsa G. Shapiro et al.   MOLECULAR GENETICS AND METABOLISM

Intravenous infusion of iduronidase-IgG and its impact on the central nervous system in children with Hurler syndrome

(2017) Roberto Giugliani et al.   MOLECULAR GENETICS AND METABOLISM

Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III

(2017) Arunabha Ghosh et al.   Orphanet Journal of Rare Diseases

Nanotechnology-based strategies for treatment of ocular disease

(2017) Yuhua Weng et al.   Acta Pharmaceutica Sinica B

Nanotechnology applied to treatment of mucopolysaccharidoses

(2016) Roselena S. Schuh et al.   Expert Opinion on Drug Delivery

Nanomedicine as a non-invasive strategy for drug delivery across the blood brain barrier

(2016) Vivienne H. Tam et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

A strategy for soluble overexpression and biochemical characterization of halo-thermotolerant Bacillus laccase in modified E. coli

(2016) Azam Safary et al.   JOURNAL OF BIOTECHNOLOGY

Design and rationale of the study programs for BMN 250, a novel enzyme replacement therapy (ERT) for Sanfilippo syndrome type B

(2016) Adam J. Shaywitz et al.   MOLECULAR GENETICS AND METABOLISM

Cervical cord compression in mucopolysaccharidosis VI (MPS VI): Findings from the MPS VI Clinical Surveillance Program (CSP)

(2016) Guirish A. Solanki et al.   MOLECULAR GENETICS AND METABOLISM

Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction

(2016) Priya S. Kishnani 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

Targeted Polymeric Nanoparticles for Brain Delivery of High Molecular Weight Molecules in Lysosomal Storage Disorders

(2016) Marika Salvalaio et al.   PLoS One

Targeting Receptor-Mediated Transport for Delivery of Biologics Across the Blood-Brain Barrier

(2015) Jason M. Lajoie et al.   Annual Review of Pharmacology and Toxicology

A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II

(2015) Joseph Muenzer et al.   GENETICS IN MEDICINE

Site-Specific PEGylation of Therapeutic Proteins

(2015) Jonathan Dozier et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

PEGylated cationic nanoemulsions can efficiently bind and transfect pIDUA in a mucopolysaccharidosis type I murine model

(2015) Michelle Fraga et al.   JOURNAL OF CONTROLLED RELEASE

Safety and efficacy of enzyme replacement therapy with idursulfase beta in children aged younger than 6years with Hunter syndrome

(2015) Young Bae Sohn et al.   MOLECULAR GENETICS AND METABOLISM

Therapies for the bone in mucopolysaccharidoses

(2015) Shunji Tomatsu et al.   MOLECULAR GENETICS AND METABOLISM

Altered Clathrin-Independent Endocytosis in Type A Niemann-Pick Disease Cells and Rescue by ICAM-1-Targeted Enzyme Delivery

(2015) Jeff Rappaport et al.   MOLECULAR PHARMACEUTICS

RTB Lectin: a novel receptor-independent delivery system for lysosomal enzyme replacement therapies

(2015) Walter Acosta et al.   Scientific Reports

Combination-targeting to multiple endothelial cell adhesion molecules modulates binding, endocytosis, and in vivo biodistribution of drug nanocarriers and their therapeutic cargoes

(2014) Iason Papademetriou et al.   JOURNAL OF CONTROLLED RELEASE

Site-specific PEGylation of Proteins: Recent Developments

(2014) Nicole Nischan et al.   JOURNAL OF ORGANIC CHEMISTRY

SBC-103, a recombinant human alpha-N-acetylglucosaminidase, demonstrates mannose-6-phosphate receptor dependent transport in an in vitro blood-brain barrier model

(2014) Anthony Rossomando et al.   MOLECULAR GENETICS AND METABOLISM

Laronidase-Functionalized Multiple-Wall Lipid-Core Nanocapsules: Promising Formulation for a More Effective Treatment of Mucopolysaccharidosis Type I

(2014) Fabiana Quoos Mayer et al.   PHARMACEUTICAL RESEARCH

Prodrugs: A challenge for the drug development

(2014) Jolanta B. Zawilska et al.   Pharmacological Reports

Intrathecal delivery of protein therapeutics to the brain: A critical reassessment

(2014) Pericles Calias et al.   PHARMACOLOGY & THERAPEUTICS

IgE-mediated anaphylaxis and allergic reactions to idursulfase in patients with Hunter syndrome

(2013) J. Kim et al.   ALLERGY

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

Enhancing Biodistribution of Therapeutic Enzymes In Vivo by Modulating Surface Coating and Concentration of ICAM-1-Targeted Nanocarriers

(2013) Janet Hsu et al.   Journal of Biomedical Nanotechnology

Early initiation of enzyme replacement therapy for the mucopolysaccharidoses

(2013) Joseph Muenzer   MOLECULAR GENETICS AND METABOLISM

Mucopolysaccharidoses and Other Lysosomal Storage Diseases

(2013) Christina Lampe et al.   RHEUMATIC DISEASE CLINICS OF NORTH AMERICA

Mucopolysaccharidoses

(2013) Rolando Cimaz et al.   Current Rheumatology Reports

Enzyme Replacement Therapy for Lysosomal Diseases: Lessons from 20 Years of Experience and Remaining Challenges

(2012) R.J. Desnick et al.   Annual Review of Genomics and Human Genetics

Intercellular Adhesion Molecule 1 Engagement Modulates Sphingomyelinase and Ceramide, Supporting Uptake of Drug Carriers by the Vascular Endothelium

(2012) Daniel Serrano et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

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

Intraperitoneal implant of recombinant encapsulated cells overexpressing alpha-l-iduronidase partially corrects visceral pathology in mucopolysaccharidosis type I mice

(2012) Guilherme Baldo et al.   CYTOTHERAPY

Mucopolysacccharidoses: from understanding to treatment, a century of discoveries

(2012) Roberto Giugliani   GENETICS AND MOLECULAR BIOLOGY

Comparative binding, endocytosis, and biodistribution of antibodies and antibody-coated carriers for targeted delivery of lysosomal enzymes to ICAM-1 versus transferrin receptor

(2012) Jason Papademetriou et al.   JOURNAL OF INHERITED METABOLIC DISEASE

CNS Penetration of Intrathecal-Lumbar Idursulfase in the Monkey, Dog and Mouse: Implications for Neurological Outcomes of Lysosomal Storage Disorder

(2012) Pericles Calias et al.   PLoS One

Production of α-L-iduronidase in maize for the potential treatment of a human lysosomal storage disease

(2012) Xu He et al.   Nature Communications

Novel water-soluble polyurethane nanomicelles for cancer chemotherapy: physicochemical characterization and cellular activities

(2012) Ahmad Khosroushahi et al.   JOURNAL OF NANOBIOTECHNOLOGY

Recombinant Encapsulated Cells Overexpressing Alpha-L-Iduronidase Correct Enzyme Deficiency in Human Mucopolysaccharidosis Type I Cells

(2011) Guilherme Baldo et al.   CELLS TISSUES ORGANS

Cell-and Gene-based Therapeutic Approaches for Neurological Deficits in Mucopolysaccharidoses

(2011) Dao Pan   CURRENT PHARMACEUTICAL BIOTECHNOLOGY

The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: Lessons learned from infantile Pompe disease

(2011) Suhrad G. Banugaria et al.   GENETICS IN MEDICINE

Bisphosphonate-decorated lipid nanoparticles designed as drug carriers for bone diseases

(2011) Guilin Wang et al.   JOURNAL OF BIOMEDICAL MATERIALS RESEARCH PART A

Biodistribution and pharmacodynamics of recombinant human alpha-l-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations

(2011) Charles H. Vite et al.   MOLECULAR GENETICS AND METABOLISM

Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle

(2011) Dwight D. Koeberl et al.   MOLECULAR GENETICS AND METABOLISM

Mannose-6-phosphate pathway: A review on its role in lysosomal function and dysfunction

(2011) Maria Francisca Coutinho et al.   MOLECULAR GENETICS AND METABOLISM

Enhanced delivery of α-glucosidase for Pompe disease by ICAM-1-targeted nanocarriers: comparative performance of a strategy for three distinct lysosomal storage disorders

(2011) Janet Hsu et al.   Nanomedicine-Nanotechnology Biology and Medicine

Pathogenesis of skeletal and connective tissue involvement in the mucopolysaccharidoses: glycosaminoglycan storage is merely the instigator

(2011) L. A. Clarke   RHEUMATOLOGY

Therapy for the mucopolysaccharidoses

(2011) V. Valayannopoulos et al.   RHEUMATOLOGY

Overview of the mucopolysaccharidoses

(2011) J. Muenzer   RHEUMATOLOGY

Anatomy and physiology of the human eye: effects of mucopolysaccharidoses disease on structure and function - a review

(2010) Colin E Willoughby et al.   CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY

Lysosomal fusion and SNARE function are impaired by cholesterol accumulation in lysosomal storage disorders

(2010) Alessandro Fraldi et al.   EMBO JOURNAL

Pharmacokinetics and tolerance study of intravitreal injection of dexamethasone-loaded nanoparticles in rabbits

(2010) Linhua Zhang   International Journal of Nanomedicine

Enhanced endothelial delivery and biochemical effects of α-galactosidase by ICAM-1-targeted nanocarriers for Fabry disease

(2010) Janet Hsu et al.   JOURNAL OF CONTROLLED RELEASE

Biopharmaceutical drug targeting to the brain

(2010) William M. Pardridge   JOURNAL OF DRUG TARGETING

Synthesis and In Vitro Release of Adriamycin from Star-Shaped Poly(Lactide-co-Glycolide) Nano- and Microparticles

(2010) Mohammad Mooguee et al.   JOURNAL OF PHARMACEUTICAL SCIENCES

Synthesis and in vitro Studies of Cross-Linked Hydrogel Nanoparticles Containing Amoxicillin

(2010) Mohammad Moogooee et al.   JOURNAL OF PHARMACEUTICAL SCIENCES

Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I

(2010) Patricia I. Dickson et al.   MOLECULAR GENETICS AND METABOLISM

New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders

(2010) Silvia Muro   Wiley Interdisciplinary Reviews-Nanomedicine and Nanobiotechnology

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

(2009) Priya S. Kishnani et al.   MOLECULAR GENETICS AND METABOLISM

Brain targeting PBCA nanoparticles and the blood–brain barrier

(2009) B Wilson   Nanomedicine

Ocular novel drug delivery: impacts of membranes and barriers

(2008) Jaleh Barar et al.   Expert Opinion on Drug Delivery

Effect of high dose, repeated intra-cerebrospinal fluid injection of sulphamidase on neuropathology in mucopolysaccharidosis type IIIA mice

(2008) K. M. Hemsley et al.   GENES BRAIN AND BEHAVIOR

Delivery of Acid Sphingomyelinase in Normal and Niemann-Pick Disease Mice Using Intercellular Adhesion Molecule-1-Targeted Polymer Nanocarriers

(2008) C. Garnacho et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

Mannose 6-Phosphate Receptor–mediated Transport of Sulfamidase Across the Blood–brain Barrier in the Newborn Mouse

(2008) Akihiko Urayama et al.   MOLECULAR THERAPY

Endothelial targeting of semi-permeable polymer nanocarriers for enzyme therapies

(2007) Thomas D. Dziubla et al.   BIOMATERIALS