Abstract
Lysosomal storage disorders (LSD) arise from genetic deficiency of a lysosomal enzyme (or its transport) and from subsequent accumulation of the enzyme substrates. Haemopoietic Stem Cell Transplant (HSCT) therapy for LSD corrects disease through cross correction of enzyme deficiency in recipient cells by enzyme secreted from engrafted, donor blood cells. The process of such transplant, its efficacy and its inherent risks are central in defining its current place in therapy. Two decades of HSCT have seen dramatically improving results as well as an improved understanding of the factors that affect outcome in both the short and long term. With improving survival figures transplant might become a viable treatment for patients that were traditionally excluded from transplant because of perceived risk. Some LSD respond better to HSCT than others and even within a responding disease some organs respond better than others. We develop the concept of delivered enzyme following cellular and other therapies of LSDs, including pharmacological enzyme replacement therapy (ERT). Delivered enzyme must be sufficient to achieve a correctable threshold that is both tissue and disease specific. Therapies of LSDs in the future will be more effective as they improve enzyme delivery to tissues and do so more safely and with reduced long term toxicities.
Current Pediatric Reviews
Title: Cellular Therapy of Lysosomal Storage Disorders: Current Status and Future Prospects
Volume: 5 Issue: 3
Author(s): Robert F. Wynn, Matthew Stubbs, Nurdan Ozyilmaz, J. Ed Wraith and Brian Bigger
Affiliation:
Abstract: Lysosomal storage disorders (LSD) arise from genetic deficiency of a lysosomal enzyme (or its transport) and from subsequent accumulation of the enzyme substrates. Haemopoietic Stem Cell Transplant (HSCT) therapy for LSD corrects disease through cross correction of enzyme deficiency in recipient cells by enzyme secreted from engrafted, donor blood cells. The process of such transplant, its efficacy and its inherent risks are central in defining its current place in therapy. Two decades of HSCT have seen dramatically improving results as well as an improved understanding of the factors that affect outcome in both the short and long term. With improving survival figures transplant might become a viable treatment for patients that were traditionally excluded from transplant because of perceived risk. Some LSD respond better to HSCT than others and even within a responding disease some organs respond better than others. We develop the concept of delivered enzyme following cellular and other therapies of LSDs, including pharmacological enzyme replacement therapy (ERT). Delivered enzyme must be sufficient to achieve a correctable threshold that is both tissue and disease specific. Therapies of LSDs in the future will be more effective as they improve enzyme delivery to tissues and do so more safely and with reduced long term toxicities.
Export Options
About this article
Cite this article as:
Wynn F. Robert, Stubbs Matthew, Ozyilmaz Nurdan, Wraith Ed J. and Bigger Brian, Cellular Therapy of Lysosomal Storage Disorders: Current Status and Future Prospects, Current Pediatric Reviews 2009; 5 (3) . https://dx.doi.org/10.2174/157339609789007187
DOI https://dx.doi.org/10.2174/157339609789007187 |
Print ISSN 1573-3963 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-6336 |
Call for Papers in Thematic Issues
Early-life nutrition: impacts on health and diseases
In early life, organs and hormonal axes are especially susceptible to changes that can affect the intestinal mucosa, immune system, and long-term health. Critical factors in the developmental process, including maternal diet, type of delivery, breastfeeding, and the nutritional status of mothers and children, have gained prominence. Evidence suggests that ...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
-
The Effect of GLUT1 on the Survival Rate and Immune Cell Infiltration of Lung
Adenocarcinoma and Squamous Cell Carcinoma: A Meta and Bioinformatics
Analysis
Anti-Cancer Agents in Medicinal Chemistry Type 2 Diabetes and Heart Failure: Challenges and Solutions
Current Cardiology Reviews The Role of TRIM Proteins in Vascular Disease
Current Vascular Pharmacology Matrix Metalloproteinases: Drug Targets for Myocardial Infarction
Current Drug Targets Pathophysiology and Treatment of Obesity Hypertension
Current Pharmaceutical Design Curcumin: A Natural Product for Diabetes and its Complications
Current Topics in Medicinal Chemistry Thyroid Disorders in Chronic Heart Failure: From Prognostic Set-up to Therapeutic Management
Endocrine, Metabolic & Immune Disorders - Drug Targets Mitochondrial and Nuclear Genes of Mitochondrial Components in Cancer
Current Genomics Cellular Mechanisms for Diastolic Dysfunction in the Human Heart
Current Pharmaceutical Biotechnology Valve Endothelial Cells – Not Just Any Old Endothelial Cells
Current Vascular Pharmacology Polyamines and Related Nitrogen Compounds in the Chemotherapy of Neglected Diseases Caused by Kinetoplastids
Current Topics in Medicinal Chemistry Patent Selections :
Recent Patents on Cardiovascular Drug Discovery Amniotic Fluid Embolism: Review
Current Pharmaceutical Biotechnology Effect of β-Blockers on Perioperative Myocardial Ischemia in Patients Undergoing Noncardiac Surgery
Current Drug Targets Anti-allergic Assessment of Ethanol Extractives of <i>Quisqualis Indica</i> Linn
Current Bioactive Compounds Cellular Therapy for Heart Failure
Current Cardiology Reviews The Right Ventricle: Biologic Insights and Response to Disease
Current Cardiology Reviews Novel Insights into Complex Cardiovascular Pathologies using 4D Flow Analysis by Cardiovascular Magnetic Resonance Imaging
Current Pharmaceutical Design Coenzyme Q10 in Cardiovascular and Metabolic Diseases: Current State of the Problem
Current Cardiology Reviews Choosing the Adaptive Cardiac Phase for Assessing Cardiac Dimensions Using Cardiac Computed Tomography for Heart Disease
Current Medical Imaging