Abstract
X-linked agammaglobulinemia (XLA), or Brutons disease, is the most common human primary humoral immunodeficiency. XLA is caused by mutations of the Brutons tyrosine kinase (BTK), a key regulator of B-cell physiology. Since the mid 80s, substitutive therapy by intravenous gammaglobulin infusions has significantly improved XLA patient survival and quality of life. Nevertheless, some frequent affections persist despite treatment, and lead to handicapping and further to morbid clinical complications for XLA individuals. Development of gene therapy by transfer of the BTK gene into hematopoietic progenitors could represent an alternative strategy for the treatment of Brutons disease, with the advantage of a definitive cure for XLA patients. Gene therapy of XLA could be considered as a paradigm for future expansion of gene therapy approaches for many other diseases, since future utilization may be strictly dependent on a marked improvement of risk-benefit ratio compared to pre-existing treatments.
Keywords: Bruton's disease, BTK, Immunodeficiency, B-cell, Retroviral vector, Hematopoietic stem cell
Current Gene Therapy
Title: Potential Application of Gene Therapy to X-Linked Agammaglobulinemia
Volume: 7 Issue: 4
Author(s): Thomas Moreau, Boris Calmels, Vincent Barlogis, Gerard Michel, Cecile Tonnelle and Christian Chabannon
Affiliation:
Keywords: Bruton's disease, BTK, Immunodeficiency, B-cell, Retroviral vector, Hematopoietic stem cell
Abstract: X-linked agammaglobulinemia (XLA), or Brutons disease, is the most common human primary humoral immunodeficiency. XLA is caused by mutations of the Brutons tyrosine kinase (BTK), a key regulator of B-cell physiology. Since the mid 80s, substitutive therapy by intravenous gammaglobulin infusions has significantly improved XLA patient survival and quality of life. Nevertheless, some frequent affections persist despite treatment, and lead to handicapping and further to morbid clinical complications for XLA individuals. Development of gene therapy by transfer of the BTK gene into hematopoietic progenitors could represent an alternative strategy for the treatment of Brutons disease, with the advantage of a definitive cure for XLA patients. Gene therapy of XLA could be considered as a paradigm for future expansion of gene therapy approaches for many other diseases, since future utilization may be strictly dependent on a marked improvement of risk-benefit ratio compared to pre-existing treatments.
Export Options
About this article
Cite this article as:
Thomas Moreau , Boris Calmels , Vincent Barlogis , Gerard Michel , Cecile Tonnelle and Christian Chabannon , Potential Application of Gene Therapy to X-Linked Agammaglobulinemia, Current Gene Therapy 2007; 7 (4) . https://dx.doi.org/10.2174/156652307781369128
DOI https://dx.doi.org/10.2174/156652307781369128 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
Call for Papers in Thematic Issues
Programmed Cell Death Genes in Oncology: Pioneering Therapeutic and Diagnostic Frontiers (BMS-CGT-2024-HT-45)
Programmed Cell Death (PCD) is recognized as a pivotal biological mechanism with far-reaching effects in the realm of cancer therapy. This complex process encompasses a variety of cell death modalities, including apoptosis, autophagic cell death, pyroptosis, and ferroptosis, each of which contributes to the intricate landscape of cancer development and ...read more
Related Journals
- 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
- Announcements
Related Articles
-
Central Nervous System-Related
Current Bioactive Compounds ADAMTS Proteinases: Potential Therapeutic Targets?
Current Pharmaceutical Biotechnology Therapeutic Targeting of Chemokines and Chemokine Receptors in Multiple Sclerosis: Opportunities and Challenges
CNS & Neurological Disorders - Drug Targets Molecular Approach to Targeted Therapy for Multiple Sclerosis
CNS & Neurological Disorders - Drug Targets Poly (ADP-Ribose) Polymerase-1 (PARP-1) as Immune Regulator
Endocrine, Metabolic & Immune Disorders - Drug Targets Melatonin as a Therapeutic Resource for Inflammatory Visual Diseases
Current Neuropharmacology Human Use of Leucoselect® Phytosome® with Special Reference to Inflammatory- Allergic Pathologies in Frail Elderly Patients
Current Pharmaceutical Design Proinflammatory Activities of Leptin in Non-Autoimmune Conditions
Inflammation & Allergy - Drug Targets (Discontinued) Understanding Heterogeneity in Supplementation Effects of Selenium in Men: A Study of Stratification Variables and Human Genetics in a Prospective Sample from New Zealand
Current Pharmacogenomics and Personalized Medicine 2 Receptor Specific Ligand Conjugated Nanocarriers: An Effective Strategy for Targeted Therapy of Tuberculosis
Current Drug Delivery From Bone Marrow Transplantation to Cellular Therapies: Possible Therapeutic Strategies in Managing Autoimmune Disorders
Current Pharmaceutical Design Endothelial Chemokines in Autoimmune Disease
Current Pharmaceutical Design CypD: The Key to the Death Door
CNS & Neurological Disorders - Drug Targets Down Regulated Expression of Claudin-1 and Claudin-5 and Up Regulation of β-Catenin: Association with Human Glioma Progression
CNS & Neurological Disorders - Drug Targets Proteomic Analysis of Phosphorylation in the Brain
Current Proteomics Role of the Innate Immune System in Autoimmune Inflammatory Demyelination
Current Medicinal Chemistry Macrophage Heterogeneity: Relevance and Functional Implications in Atherosclerosis
Current Vascular Pharmacology JAK Inhibitors: Pharmacology and Clinical Activity in Chronic Myeloprolipherative Neoplasms
Current Medicinal Chemistry B Cells: Programmers of CD4 T Cell Responses
Infectious Disorders - Drug Targets Significance of P2X7 Receptor Variants to Human Health and Disease
Recent Patents on DNA & Gene Sequences