Abstract
Mucopolysaccharidoses (MPS) are a group of lysosomal storage diseases that are resulted from abnormal accumulation of glycosaminoglycans. Among the progressive multi-organ abnormalities often associated with MPS diseases, the deterioration of central nervous system (CNS) is the most challenging manifestations to be tackled, due to the impermeability of the blood-brain-barrier (BBB). Evolved with recent development in stem cell biotechnology and gene therapy, several novel experimental approaches have been investigated in animal models. In this review, we will address different approaches attempting to bypass the BBB for neuropathic MPS treatment using cell- and gene-based therapies. Several neurological findings in CNS pathophysiology emerged with therapeutic investigation will also be discussed.
Keywords: Mucopolysaccharidoses, central nervous system, blood-brain-barrier, animal models, stem cells, gene therapy, abnormal accumulation of glycosaminoglycans, multi-organ abnormalities, deterioration of central nervous system, dysostosis multiplex, Hurler syndrome, enzyme trafficking, lysosomal enzyme release and reuptake, hematopoietic stem cell (HSC) transplantation
Current Pharmaceutical Biotechnology
Title: Cell-and Gene-based Therapeutic Approaches for Neurological Deficits in Mucopolysaccharidoses
Volume: 12 Issue: 6
Author(s): Dao Pan
Affiliation:
Keywords: Mucopolysaccharidoses, central nervous system, blood-brain-barrier, animal models, stem cells, gene therapy, abnormal accumulation of glycosaminoglycans, multi-organ abnormalities, deterioration of central nervous system, dysostosis multiplex, Hurler syndrome, enzyme trafficking, lysosomal enzyme release and reuptake, hematopoietic stem cell (HSC) transplantation
Abstract: Mucopolysaccharidoses (MPS) are a group of lysosomal storage diseases that are resulted from abnormal accumulation of glycosaminoglycans. Among the progressive multi-organ abnormalities often associated with MPS diseases, the deterioration of central nervous system (CNS) is the most challenging manifestations to be tackled, due to the impermeability of the blood-brain-barrier (BBB). Evolved with recent development in stem cell biotechnology and gene therapy, several novel experimental approaches have been investigated in animal models. In this review, we will address different approaches attempting to bypass the BBB for neuropathic MPS treatment using cell- and gene-based therapies. Several neurological findings in CNS pathophysiology emerged with therapeutic investigation will also be discussed.
Export Options
About this article
Cite this article as:
Pan Dao, Cell-and Gene-based Therapeutic Approaches for Neurological Deficits in Mucopolysaccharidoses, Current Pharmaceutical Biotechnology 2011; 12 (6) . https://dx.doi.org/10.2174/138920111795542679
DOI https://dx.doi.org/10.2174/138920111795542679 |
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
-
Does Caffeine Affect Cardiovascular Responses?
Vascular Disease Prevention (Discontinued) A Stress Repair Mechanism That Maintains Vertebrate Structure During Stress
Cardiovascular & Hematological Disorders-Drug Targets Phycobilins and Phycobiliproteins Used in Food Industry and Medicine
Mini-Reviews in Medicinal Chemistry Minocycline Repurposing in Critical Illness: Focus on Stroke
Current Topics in Medicinal Chemistry Obesity and Cardioprotection
Current Medicinal Chemistry Cardiac Metabolism in Myocardial Ischemia
Current Pharmaceutical Design Effects of Coenzyme Q and Creatine Supplementation on Brain Energy Metabolism in Rats Exposed to Chronic Cerebral Hypoperfusion
Current Alzheimer Research Drug-Eluting Stents: Present and Future
Cardiovascular & Hematological Agents in Medicinal Chemistry The Use of Genomics and Proteomics for the Recognition of Transplantation Rejection of Solid Organs
Recent Patents on DNA & Gene Sequences Recent Developments on Endothelin Antagonists as Immunomodulatory Drugs - from Infection to Transplantation Medicine
Recent Patents on Cardiovascular Drug Discovery Regulation of the Neuronal Fate by ΔFosB and its Downstream Target, Galectin-1
Current Drug Targets HSP70 Family in the Renal Inflammatory Response
Inflammation & Allergy - Drug Targets (Discontinued) Antioxidative Peptides: Trends and Perspectives for Future Research
Current Medicinal Chemistry Diabetes and Its Complications: Therapies Available, Anticipated and Aspired
Current Diabetes Reviews Advances in Antithrombotic Agents
Cardiovascular & Hematological Agents in Medicinal Chemistry Endothelial Effects of Drugs Designed to Treat Erectile Dysfunction
Current Pharmaceutical Design Cardiac Oxidative Stress and Inflammatory Cytokines Response after Myocardial Infarction
Current Vascular Pharmacology Pharmaceutical Polymeric Nanoparticles Prepared by the Double Emulsion- Solvent Evaporation Technique
Recent Patents on Drug Delivery & Formulation Phosphoinositide 3-Kinase Gamma (PI3Kγ) Inhibitors for the Treatment of Inflammation and Autoimmune Disease
Recent Patents on Inflammation & Allergy Drug Discovery Cardioprotection Acquired Through Exercise: The Role of Ischemic Preconditioning
Current Cardiology Reviews