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 |
- 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
-
Written Consent to Use the Drug in Children: The Problem of Off-Label Drugs
Current Pharmaceutical Design Statins and Oxidative Stress in the Cardiovascular System
Current Pharmaceutical Design Why Multiples of 21? Why does Selenoprotein P Contain Multiple Selenocysteine Residues?
Current Nutraceuticals Caring for HIV-Infected Patients in the ICU in The Highly Active Antiretroviral Therapy Era
Current HIV Research Cord Blood as Diagnostic Window for Preeclampsia
Recent Patents on Biomarkers Potential Roles of MyomiRs in Cardiac Development and Related Diseases
Current Cardiology Reviews Cardiac Regeneration: Stem Cells and Beyond
Current Medicinal Chemistry Targeting the Toll-System in Cardiovascular Sciences
Recent Patents on Inflammation & Allergy Drug Discovery Prospectives of Antihypertensive Nano-ceuticals as Alternative Therapeutics
Current Drug Targets Beneficial Extracardiac Effects of Cardiovascular Medications
Current Cardiology Reviews Targeting SUMOylation Cascade for Diabetes Management
Current Drug Targets Oxidative Stress and Endothelial Dysfunction in Cardiovascular Disease: Mitochondria-Targeted Therapeutics
Current Medicinal Chemistry Therapeutic Applications of Human Heme Oxygenase Gene Transfer and Gene Therapy
Current Pharmaceutical Design Na+,K+-ATPase as a Target for Treatment of Tissue Fibrosis
Current Medicinal Chemistry Selenoproteins and their Role in Oxidative Stress and Inflammation
Current Chemical Biology Cruzipain, the Major Cysteine Protease of Trypanosoma cruzi: A Sulfated Glycoprotein Antigen as Relevant Candidate for Vaccine Development and Drug Target. A Review
Current Medicinal Chemistry Nitric Oxide: State of the Art in Drug Design
Current Medicinal Chemistry Vitamins B1, B2, B3 and B9 – Occurrence, Biosynthesis Pathways and Functions in Human Nutrition
Mini-Reviews in Medicinal Chemistry Cardiac and Muscular Involvement in Idiopathic Inflammatory Myopathies: Noninvasive Diagnostic Assessment and the Role of Cardiovascular and Skeletal Magnetic Resonance Imaging
Inflammation & Allergy - Drug Targets (Discontinued) Exercise and Stevia Rebaudiana (R) Extracts Attenuate Diabetic Cardiomyopathy in Type 2 Diabetic Rats: Possible Underlying Mechanisms
Endocrine, Metabolic & Immune Disorders - Drug Targets