Abstract
Mucopolysaccharosis III (MPS III) is a lysosomal storage disorder and belongs to the group of mucopolysaccharidoses. MPS III is caused by a deficiency of one of the four enzymes catalyzing the degradation of the glycosaminoglycan heparan sulfate. MPS III is clinically characterized by progressive dementia with distinct behavioral disturbances and relatively mild somatic disease. This review will summarize and discuss the available and potential future therapeutic options for patients with MPS III. This includes enzyme replacement therapy (ERT), hematopoietic stem cell transplantation (HSCT), substrate reduction therapy (SRT), chaperone-mediated therapy, and gene therapy. Although clinical efficacy has not yet been fully demonstrated for any of these therapies, it is likely that future developments will lead to disease- modifying treatment for this devastating disease.
Keywords: Enzyme replacement therapy, gene therapy, hematopoietic stem cell transplantation, mucopolysaccharidosis iii, sanfilippo syndrome, substrate reduction therapy, lysosomal storage disorder, glycosaminoglycan heparan sulfate, behavioral disturbances, hematopoietic stem cell transplantation (HSCT), chaperone-mediated therapy, disease-modifying treatmen, autosomal recessive inherited deficiency
Current Pharmaceutical Biotechnology
Title: Mucopolysaccharidosis Type III (Sanfilippo Syndrome): Emerging Treatment Strategies
Volume: 12 Issue: 6
Author(s): J. de Ruijter, M. J. Valstar and F. A. Wijburg
Affiliation:
Keywords: Enzyme replacement therapy, gene therapy, hematopoietic stem cell transplantation, mucopolysaccharidosis iii, sanfilippo syndrome, substrate reduction therapy, lysosomal storage disorder, glycosaminoglycan heparan sulfate, behavioral disturbances, hematopoietic stem cell transplantation (HSCT), chaperone-mediated therapy, disease-modifying treatmen, autosomal recessive inherited deficiency
Abstract: Mucopolysaccharosis III (MPS III) is a lysosomal storage disorder and belongs to the group of mucopolysaccharidoses. MPS III is caused by a deficiency of one of the four enzymes catalyzing the degradation of the glycosaminoglycan heparan sulfate. MPS III is clinically characterized by progressive dementia with distinct behavioral disturbances and relatively mild somatic disease. This review will summarize and discuss the available and potential future therapeutic options for patients with MPS III. This includes enzyme replacement therapy (ERT), hematopoietic stem cell transplantation (HSCT), substrate reduction therapy (SRT), chaperone-mediated therapy, and gene therapy. Although clinical efficacy has not yet been fully demonstrated for any of these therapies, it is likely that future developments will lead to disease- modifying treatment for this devastating disease.
Export Options
About this article
Cite this article as:
de Ruijter J., J. Valstar M. and A. Wijburg F., Mucopolysaccharidosis Type III (Sanfilippo Syndrome): Emerging Treatment Strategies, Current Pharmaceutical Biotechnology 2011; 12 (6) . https://dx.doi.org/10.2174/138920111795542651
DOI https://dx.doi.org/10.2174/138920111795542651 |
Print ISSN 1389-2010 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-4316 |
Call for Papers in Thematic Issues
Artificial Intelligence in Bioinformatics
Bioinformatics is an interdisciplinary field that analyzes and explores biological data. This field combines biology and information system. Artificial Intelligence (AI) has attracted great attention as it tries to replicate human intelligence. It has become common technology for analyzing and solving complex data and problems and encompasses sub-fields of machine ...read more
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
Related Articles
-
Hemolysis-Associated Pulmonary Hypertension in Sickle Cell Disease: Global Disruption of the Arginine-Nitric Oxide Pathway
Current Hypertension Reviews Targeting Stress Activated Protein Kinases, JNK and p38, as New Therapeutic Approach for Neurodegenerative Diseases
Central Nervous System Agents in Medicinal Chemistry Plants Indicated by Brazilian Indians for Disturbances of the Central Nervous System: A Bibliographical Survey
Central Nervous System Agents in Medicinal Chemistry The Role of Nitric Oxide Synthase Inhibitors in Schizophrenia
Current Medicinal Chemistry Early β-Amyloid-induced Synaptic Dysfunction Is Counteracted by Estrogen in Organotypic Hippocampal Cultures
Current Alzheimer Research Lentiviral Vector-Based Models of Amyloid Pathology: From Cells to Animals
Current Alzheimer Research The Immunoproteasome: An Emerging Therapeutic Target
Current Topics in Medicinal Chemistry Pharmacogenomics – Implications in the Development of HIV-Associated Brain Disease
Anti-Inflammatory & Anti-Allergy Agents in Medicinal Chemistry Neural Basis of Anhedonia and Amotivation in Patients with Schizophrenia: The Role of Reward System
Current Neuropharmacology A Dual Activity of ROS and Oxidative Stress on Adult Neurogenesis and Alzheimers Disease
Central Nervous System Agents in Medicinal Chemistry Visual Spatial Attention Training Improve Spatial Attention and Motor Control for Unilateral Neglect Patients
CNS & Neurological Disorders - Drug Targets Endocannabinoid System: Emerging Role from Neurodevelopment to Neurodegeneration
Mini-Reviews in Medicinal Chemistry Evaluation on Monocyte-Mediated Delivery of a Therapeutic Gene into the Inflamed Brain
Current Gene Therapy 3D MI-DRAGON: New Model for the Reconstruction of US FDA Drug- Target Network and Theoretical-Experimental Studies of Inhibitors of Rasagiline Derivatives for AChE
Current Topics in Medicinal Chemistry The Role of miR-124 in Drosophila Alzheimer's Disease Model by Targeting Delta in Notch Signaling Pathway
Current Molecular Medicine Inflammatory Cyclooxygenase Activity and PGE<sub>2</sub> Signaling in Models of Alzheimer’s Disease
Current Immunology Reviews (Discontinued) Chronic Infusion of Amyloid-β Peptide and Sustained Attention Altered α7 Nicotinic Receptor Density in the Rat Brain
Current Alzheimer Research Rikkunshito and Ghrelin Secretion
Current Pharmaceutical Design Inflammation, Oxidative Stress and Metabolic Syndrome: Dietary Modulation
Current Vascular Pharmacology Coumarins: Auspicious Cholinesterase and Monoamine Oxidase Inhibitors
Current Topics in Medicinal Chemistry