- Home
- Publications
- Publication Search
- Publication Details
Title
How close are we to therapies for Sanfilippo disease?
Authors
Keywords
-
Journal
METABOLIC BRAIN DISEASE
Volume 33, Issue 1, Pages 1-10
Publisher
Springer Nature
Online
2017-09-18
DOI
10.1007/s11011-017-0111-4
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID
- (2017) Carles Roca et al. HUMAN MOLECULAR GENETICS
- Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration
- (2017) Adeline A. Lau et al. JOURNAL OF MOLECULAR MEDICINE-JMM
- Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial
- (2017) Marc Tardieu et al. LANCET NEUROLOGY
- 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
- Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery
- (2017) Haiyan Fu et al. MOLECULAR THERAPY
- Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III
- (2017) Arunabha Ghosh et al. Orphanet Journal of Rare Diseases
- Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice
- (2017) Mika Aoyagi-Scharber et al. Molecular Therapy-Methods & Clinical Development
- Low-dose, continual enzyme delivery ameliorates some aspects of established brain disease in a mouse model of a childhood-onset neurodegenerative disorder
- (2016) Barbara King et al. EXPERIMENTAL NEUROLOGY
- Emerging drugs for the treatment of mucopolysaccharidoses
- (2016) Roberto Giugliani et al. EXPERT OPINION ON EMERGING DRUGS
- Glycosaminoglycans and mucopolysaccharidosis type III
- (2016) Grzegorz Wegrzyn Frontiers in Bioscience-Landmark
- A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome
- (2016) Leanne K. Winner et al. HUMAN GENE THERAPY
- Low-dose, continuous enzyme replacement therapy ameliorates brain pathology in the neurodegenerative lysosomal disorder mucopolysaccharidosis type IIIA
- (2016) Barbara King et al. JOURNAL OF NEUROCHEMISTRY
- 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
- 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
- Therapeutic Potential of Hydroxypropyl-β-Cyclodextrin-Based Extract of Medicago sativa in the Treatment of Mucopolysaccharidoses
- (2016) Barbara Fumić et al. PLANTA MEDICA
- Hematopoietic Cell Transplantation for Mucopolysaccharidosis Patients Is Safe and Effective: Results after Implementation of International Guidelines
- (2015) Mieke Aldenhoven et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- The impact of HIV-1 genetic diversity on the efficacy of a combinatorial RNAi-based gene therapy
- (2015) E Herrera-Carrillo et al. GENE THERAPY
- Determination of the role of injection site on the efficacy of intra-CSF enzyme replacement therapy in MPS IIIA mice
- (2015) Helen Beard et al. MOLECULAR GENETICS AND METABOLISM
- Sanfilippo syndrome: Overall review
- (2015) Fernando Andrade et al. PEDIATRICS INTERNATIONAL
- EXTL2 and EXTL3 inhibition with siRNAs as a promising substrate reduction therapy for Sanfilippo C syndrome
- (2015) Isaac Canals et al. Scientific Reports
- A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB
- (2015) Aaron S. Meadows et al. Human Gene Therapy Clinical Development
- Evaluation of Aminoglycoside and Non-Aminoglycoside Compounds for Stop-Codon Readthrough Therapy in Four Lysosomal Storage Diseases
- (2015) Marta Gómez-Grau et al. PLoS One
- Repeated Administrations of Human Umbilical Cord Blood Cells Improve Disease Outcomes in a Mouse Model of Sanfilippo Syndrome Type III B
- (2014) Alison E. Willing et al. CELL TRANSPLANTATION
- Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human SGSH and SUMF1 cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial
- (2014) Marc Tardieu et al. HUMAN GENE THERAPY
- Biochemical, histological and functional correction of mucopolysaccharidosis Type IIIB by intra-cerebrospinal fluid gene therapy
- (2014) Albert Ribera et al. HUMAN MOLECULAR GENETICS
- Correction of murine mucopolysaccharidosis type IIIA central nervous system pathology by intracerebroventricular lentiviral-mediated gene delivery
- (2014) Chantelle McIntyre et al. JOURNAL OF GENE MEDICINE
- Evaluation of enzyme dose and dose-frequency in ameliorating substrate accumulation in MPS IIIA Huntaway dog brain
- (2014) Barbara King et al. JOURNAL OF INHERITED METABOLIC DISEASE
- 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
- Therapeutic strategies based on modified U1 snRNAs and chaperones for Sanfilippo C splicing mutations
- (2014) Liliana Matos et al. Orphanet Journal of Rare Diseases
- 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
- Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts
- (2013) Shih-hsin Kan et al. BIOCHEMICAL JOURNAL
- Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice
- (2013) C D Heldermon et al. GENE THERAPY
- Treatment effect of coenzyme Q10 and an antioxidant cocktail in fibroblasts of patients with Sanfilippo disease
- (2013) Leslie Matalonga et al. JOURNAL OF INHERITED METABOLIC DISEASE
- High dose genistein aglycone therapy is safe in patients with mucopolysaccharidoses involving the central nervous system
- (2013) Katherine H. Kim et al. MOLECULAR GENETICS AND METABOLISM
- Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathic Lysosomal Disease
- (2013) Ana Sergijenko et al. MOLECULAR THERAPY
- Gene expression-targeted isoflavone therapy
- (2012) Alicja Węgrzyn IUBMB LIFE
- Genistein Improves Neuropathology and Corrects Behaviour in a Mouse Model of Neurodegenerative Metabolic Disease
- (2010) Marcelina Malinowska et al. PLoS One
- Impairment of glycosaminoglycan synthesis in mucopolysaccharidosis type IIIA cells by using siRNA: a potential therapeutic approach for Sanfilippo disease
- (2009) Dariusz Dziedzic et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- Gene silencing of EXTL2 and EXTL3 as a substrate deprivation therapy for heparan sulphate storing mucopolysaccharidoses
- (2009) Xenia Kaidonis et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice
- (2009) Marcelina Malinowska et al. MOLECULAR GENETICS AND METABOLISM
- Sanfilippo syndrome: A mini-review
- (2008) M. J. Valstar et al. JOURNAL OF INHERITED METABOLIC DISEASE
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationAdd your recorded webinar
Do you already have a recorded webinar? Grow your audience and get more views by easily listing your recording on Peeref.
Upload Now