Restoration of MBL-deficiency: redefining the safety, efficacy and viability of MBL-substitution therapy.

Abstract

MBL-deficiency is a commonly occurring deficiency of the innate immune system, affecting a substantial part of the population and has been extensively studied. MBL appears to function as a disease modifier. The role of MBL in different conditions is context-dependent. Many clinical studies show conflicting results, which can be partially explained by different definitions of MBL-deficiency, including phenotype- and genotype-based approaches. In this review we give an overview of literature of MBL, its role in different pathologies, diseases and patient populations. We review MBL replacement studies, and discuss the potential of MBL substitution therapy. We finally suggest that new MBL substitution trials should be conducted within a predefined patient population. MBL-deficiency should be based on serum levels and confirmed by genotyping.

• Authors: M P Keizer, D Wouters, L J Schlapbach, T W Kuijpers
• Journal: Molecular immunology (Mol Immunol) Vol. 61 Issue 2 Pg. 174-84 (Oct 2014) ISSN: 1872-9142 [Electronic] England
• PMID: 25044097 (Publication Type: Journal Article, Review)
• Copyright: Copyright © 2014 Elsevier Ltd. All rights reserved.

Chemical References

• Mannose-Binding Lectin
• Recombinant Proteins
• Mannose-Binding Protein-Associated Serine Proteases

Topics

• Genetic Predisposition to Disease
• Genotype
• Humans
• Mannose-Binding Lectin (chemistry, deficiency, genetics, immunology, metabolism, therapeutic use)
• Mannose-Binding Protein-Associated Serine Proteases (chemistry, genetics, metabolism)
• Metabolism, Inborn Errors (therapy)
• Recombinant Proteins (therapeutic use)
• Treatment Outcome