Abstract
Muscular dystrophies are broadly classed as skeletal muscle disease entities of genetic origin. Accordingly, the development and application of gene therapy treatment modalities has focused on skeletal muscle gene replacement. Irrespective of this generalization, most forms of dystrophy are accompanied by progressive cardiomyopathy and cardiac involvement in muscular dystrophies is now recognized as an independent risk for patient morbidity. In this review, we summarize the available murine strains most suitable for modeling the dystrophic myocardium and discuss the use of adenoviral based vector systems as the preferred gene delivery vehicle for modulating dystrophic cardiomyopathy.
Keywords: dystrophy, dystrophic cardiomyopathy, heart, adenovirus, coxsackie virus
Current Gene Therapy
Title: Getting to the Heart of the Matter: Exploring Opportunities for Gene Therapy Treatment of Dystrophic Cardiomyopathy
Volume: 4 Issue: 2
Author(s): Steven M. Kolodziejczyk and Lynn A. Megeney
Affiliation:
Keywords: dystrophy, dystrophic cardiomyopathy, heart, adenovirus, coxsackie virus
Abstract: Muscular dystrophies are broadly classed as skeletal muscle disease entities of genetic origin. Accordingly, the development and application of gene therapy treatment modalities has focused on skeletal muscle gene replacement. Irrespective of this generalization, most forms of dystrophy are accompanied by progressive cardiomyopathy and cardiac involvement in muscular dystrophies is now recognized as an independent risk for patient morbidity. In this review, we summarize the available murine strains most suitable for modeling the dystrophic myocardium and discuss the use of adenoviral based vector systems as the preferred gene delivery vehicle for modulating dystrophic cardiomyopathy.
Export Options
About this article
Cite this article as:
Kolodziejczyk M. Steven and Megeney A. Lynn, Getting to the Heart of the Matter: Exploring Opportunities for Gene Therapy Treatment of Dystrophic Cardiomyopathy, Current Gene Therapy 2004; 4 (2) . https://dx.doi.org/10.2174/1566523043346516
DOI https://dx.doi.org/10.2174/1566523043346516 |
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
-
Periostin as a Heterofunctional Regulator of Cardiac Development and Disease
Current Genomics Between Bench and Bed Side: PI3K Inhibitors
Current Molecular Pharmacology Pathogenesis of Age-Related Cataract: A Systematic Review of Proteomic Studies
Current Proteomics The Role of Autophagy in Subarachnoid Hemorrhage: An Update
Current Neuropharmacology Chromium-Picolinate Therapy in Diabetes Care: Molecular and Subcellular Profiling Revealed a Necessity for Individual Outcome Prediction, Personalised Treatment Algorithms & New Guidelines
Infectious Disorders - Drug Targets A Review on Hematopoietic Stem Cell Treatment for Epilepsy
CNS & Neurological Disorders - Drug Targets Beta-Blockers (Carvedilol) in Children with Systemic Ventricle Systolic Dysfunction - Systematic Review and Meta-Analysis
Reviews on Recent Clinical Trials Management of Inflammation by Natural Polyphenols: A Comprehensive Mechanistic Update
Current Medicinal Chemistry Non-canonical Molecular Targets for Novel Analgesics: Intracellular Calcium and HCN Channels
Current Neuropharmacology Cardiovascular Disease Risk Among the Poor and Homeless – What We Know So Far
Current Cardiology Reviews Histological Alterations in the Testicular Tissue Induced by Sildenafil Overdoses
Drug Metabolism Letters New Strategies for Managing Anemia of Chronic Kidney Disease
Cardiovascular & Hematological Agents in Medicinal Chemistry PET Measurements of cAMP-Mediated Phosphodiesterase-4 with (R)-[11C]Rolipram
Current Radiopharmaceuticals Nuclear Imaging of Post-infarction Inflammation in Ischemic Cardiac Diseases - New Radiotracers for Potential Clinical Applications
Current Radiopharmaceuticals ACE2 and Diabetic Complications
Current Pharmaceutical Design The FDG-PET Revolution of Medical Imaging – Four Decades and Beyond
Current Molecular Imaging (Discontinued) Are the Antioxidant Properties of Carvedilol Important for the Protection of Cardiac Mitochondria?
Current Vascular Pharmacology The Emerging Roles of Leptin and Ghrelin in Cardiovascular Physiology and Pathophysiology
Current Vascular Pharmacology Recent Developments on Endothelin Antagonists as Immunomodulatory Drugs - from Infection to Transplantation Medicine
Recent Patents on Cardiovascular Drug Discovery Impact of Matrix Metalloproteinases on Atherosclerosis
Current Drug Targets