Targeting Brain Disease in MPSII: Preclinical Evaluation of IDS-Loaded PLGA Nanoparticles

Advances in nanocarriers enabled brain targeted drug delivery across blood brain barrier

(2019) Garima Sharma et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms

(2018) Hélène FE Gleitz et al.   EMBO Molecular Medicine

Hybrid nanoparticles as a new technological approach to enhance the delivery of cholesterol into the brain

(2018) Daniela Belletti et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

Efficacy of Idursulfase therapy in patients with Mucopolysaccharidosis type II who initiated enzyme replacement therapy in adult age. A systematic review of the literature

(2018) J. Pérez-López 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

Blood-Brain Delivery Methods Using Nanotechnology

(2018) Daniel Teleanu et al.   Pharmaceutics

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

Brain RNA-Seq Profiling of the Mucopolysaccharidosis Type II Mouse Model

(2017) Marika Salvalaio et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Potential of surfactant-coated nanoparticles to improve brain delivery of arylsulfatase A

(2017) Tilman Schuster et al.   JOURNAL OF CONTROLLED RELEASE

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

Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry

(2017) Joseph Muenzer et al.   Orphanet Journal of Rare Diseases

Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice

(2016) Christian Hinderer et al.   HUMAN GENE THERAPY

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

(2016) Marika Salvalaio et al.   PLoS One

Cholesterol-loaded nanoparticles ameliorate synaptic and cognitive function in Huntington's disease mice

(2015) M. Valenza et al.   EMBO Molecular Medicine

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

(2015) Joseph Muenzer et al.   GENETICS IN MEDICINE

Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II

(2015) Sung Yoon Cho et al.   Orphanet Journal of Rare Diseases

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

Insight on the fate of CNS-targeted nanoparticles. Part II: Intercellular neuronal cell-to-cell transport

(2014) Giovanni Tosi et al.   JOURNAL OF CONTROLLED RELEASE

Clinical efficacy of Enzyme Replacement Therapy in paediatric Hunter patients, an independent study of 3.5 years

(2014) Rosella Tomanin et al.   Orphanet Journal of Rare Diseases

Improvement of CNS Defects Via Continuous Intrathecal Enzyme Replacement by Osmotic Pump in Mucopolysaccharidosis Type II Mice

(2013) Young Bae Sohn et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

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

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

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

Genistein reduces glycosaminoglycan levels in a mouse model of mucopolysaccharidosis type II

(2010) A Friso et al.   BRITISH JOURNAL OF PHARMACOLOGY

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

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

Gene therapy of Hunter syndrome: Evaluation of the efficiency of muscle electro gene transfer for the production and release of recombinant iduronate-2-sulfatase (IDS)

(2008) A. Friso et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Recognition and Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome)

(2008) R. Martin et al.   PEDIATRICS