Abstract
Muscular dystrophies are heterogeneous neuromuscular disorders of inherited origin, including Duchenne muscular dystrophy (DMD). Cell-based therapies were used to promote muscle regeneration with the hope that the host cells repopulated the muscle and improved muscle function and pathology. Stem cells were preferable for therapeutic applications, due to their capacity of self-renewal and differentiative potential. In the last years, encouraging results were obtained with adult stem cells to treat muscular dystrophies. Adult stem cells were found into various tissues of the body and they were able to maintain, generate, and replace terminally differentiated cells within their own specific tissue because of cell turnover or tissue injury. Moreover, it became clear that these cells could participate into regeneration of more than just their resident organ. Here, we described multiple types of muscle and non muscle-derived myogenic stem cells, their characterization and their possible use to treat muscular dystrophies. We also underlined that most promising possibility for the management and therapy of DMD is a combination of different approaches, such as gene and stem cell therapy.
Keywords: Adult stem cells, cell therapy, DMD, gene therapy, iPS cell, muscular dystrophies, CD133+, hSM-MSCs, exon, GRMD
Current Gene Therapy
Title:The Role of Stem Cells in Muscular Dystrophies
Volume: 12 Issue: 3
Author(s): Mirella Meregalli, Andrea Farini, Federica Colleoni, Letizia Cassinelli and Yvan Torrente
Affiliation:
Keywords: Adult stem cells, cell therapy, DMD, gene therapy, iPS cell, muscular dystrophies, CD133+, hSM-MSCs, exon, GRMD
Abstract: Muscular dystrophies are heterogeneous neuromuscular disorders of inherited origin, including Duchenne muscular dystrophy (DMD). Cell-based therapies were used to promote muscle regeneration with the hope that the host cells repopulated the muscle and improved muscle function and pathology. Stem cells were preferable for therapeutic applications, due to their capacity of self-renewal and differentiative potential. In the last years, encouraging results were obtained with adult stem cells to treat muscular dystrophies. Adult stem cells were found into various tissues of the body and they were able to maintain, generate, and replace terminally differentiated cells within their own specific tissue because of cell turnover or tissue injury. Moreover, it became clear that these cells could participate into regeneration of more than just their resident organ. Here, we described multiple types of muscle and non muscle-derived myogenic stem cells, their characterization and their possible use to treat muscular dystrophies. We also underlined that most promising possibility for the management and therapy of DMD is a combination of different approaches, such as gene and stem cell therapy.
Export Options
About this article
Cite this article as:
Meregalli Mirella, Farini Andrea, Colleoni Federica, Cassinelli Letizia and Torrente Yvan, The Role of Stem Cells in Muscular Dystrophies, Current Gene Therapy 2012; 12 (3) . https://dx.doi.org/10.2174/156652312800840559
DOI https://dx.doi.org/10.2174/156652312800840559 |
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
-
Beta-Blockers use for Hypertension in the Elderly
Cardiovascular & Hematological Agents in Medicinal Chemistry TRP Channels as A Newly Emerging Non-Voltage-Gated Ca2+ Entry Channel Superfamily
Current Pharmaceutical Design Ryanodine Receptor - A Novel Therapeutic Target in Heart Disease
Recent Patents on Cardiovascular Drug Discovery Omecamtiv Mecarbil: A Myosin Motor Activator Agent with Promising Clinical Performance and New in vitro Results
Current Medicinal Chemistry Pharmacotherapy of Dilated Cardiomyopathy
Current Pharmaceutical Design Marfan Syndrome and Related Heritable Thoracic Aortic Aneurysms and Dissections
Current Pharmaceutical Design Incremental Value of Two Dimensional Speckle Tracking Echocardiography in the Functional Assessment and Characterization of Subclinical Left Ventricular Dysfunction
Current Cardiology Reviews Potential Cardio-Protective Agents: A Resveratrol Review (2000-2019)
Current Pharmaceutical Design Postponing Motherhood: A Demographic and Contemporary Issue
Current Women`s Health Reviews Re-Discovering PDE3 Inhibitors - New Opportunities for a Long Neglected Target
Current Topics in Medicinal Chemistry Alcohol and the Cardiovascular System: A Double-Edged Sword
Current Pharmaceutical Design Heart Disease in Patients with HIV/AIDS-An Emerging Clinical Problem
Current Cardiology Reviews Adipobiology for Novel Therapeutic Approaches in Metabolic Syndrome
Current Vascular Pharmacology Cardiovascular Surveillance of Duchenne and Becker Muscular Dystrophy and Female Carriers
Current Pediatric Reviews Beta-blockers in Intensive Care Medicine: Potential Benefit in Acute Brain Injury and Acute Respiratory Distress Syndrome
Recent Patents on Cardiovascular Drug Discovery Animal Mitochondria: Evolution, Function, and Disease
Current Molecular Medicine Cardiac Nuclear Imaging: Current Status and Future Directions
Current Medical Imaging Effects of Mesenchymal Stromal Cells on Diabetic Cardiomyopathy
Current Pharmaceutical Design Current Pharmacologic Management of Pediatric Heart Failure in Congenital Heart Disease
Current Vascular Pharmacology Mitochondrial Biogenesis: A Therapeutic Target for Neurodevelopmental Disorders and Neurodegenerative Diseases
Current Pharmaceutical Design