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 |
- 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
-
Improved Pharmacokinetic Profile and Anti-Inflammatory Property of a Novel Curcumin Derivative, A50
Letters in Drug Design & Discovery Recent Applications of Peanut Phytoalexins
Recent Patents on Food, Nutrition & Agriculture Effects of Alcohol in the Lung
Current Respiratory Medicine Reviews Gene-modified Mesenchymal Stem Cell–based Therapy in Renal Ischemia- Reperfusion Injury
Current Gene Therapy Vertical and Horizontal Convergences of Targeting Pathways in Combination Therapy with Baicalin and Jasminoidin for Cerebral Ischemia
CNS & Neurological Disorders - Drug Targets Regenerative Approaches to Post-Myocardial Infarction Heart Failure
Current Pharmaceutical Design Molecular Imaging of Vascular Thrombosis
Current Molecular Imaging (Discontinued) The Role of Coronary Physiology in Contemporary Percutaneous Coronary Interventions
Current Cardiology Reviews Coagulopathy as a Therapeutic Target for TRALI: Rationale and Possible Sites of Action
Current Pharmaceutical Design State of the Heart: CMR in Coronary Artery Disease
Current Medical Imaging Cerebroprotective Functions of HO-2
Current Pharmaceutical Design High Yield Immobilization and Stabilization of Oxidoreductases Using Magnetic Nanosupports and Their Potential Applications: An Update
Current Catalysis Role of AMPK in Diabetic Cardiovascular Complications: An Overview
Cardiovascular & Hematological Disorders-Drug Targets The Role of Statins in the Activation of Heme Oxygenase-1 in Cardiovascular Diseases
Current Drug Targets Carbohydrates in Therapeutics
Cardiovascular & Hematological Agents in Medicinal Chemistry Novel Trends in the Treatment of Cardiovascular Disorders: Site- and Event- Selective Adenosinergic Drugs
Current Medicinal Chemistry Strategies for Enhancing Progenitor Cell Mobilization and Function in Diabetes
Current Vascular Pharmacology Prognosis and Treatment of Ventricular Arrhythmias Following Myocardial Infarction
Current Cardiology Reviews Molecular Chaperone ORP150 in ER Stress–related Diseases
Current Pharmaceutical Design General Anesthetics in Brain Injury: Friends or Foes?
Current Pharmaceutical Design