Abstract
Limb Girdle Muscular Dystrophy (LGMD) refers to a group of 25 genetic diseases linked by common clinical features, including wasting of muscles supporting the pelvic and shoulder girdles. Cardiac involvement may also occur. Like other muscular dystrophies, LGMDs are currently incurable, but prospective gene replacement therapies targeting recessive forms have shown promise in pre-clinical and clinical studies. In contrast, little attention has been paid to developing gene therapy approaches for dominant forms of LGMD, which would likely benefit from disease gene silencing. Despite the lack of focus to date on developing gene therapies for dominant LGMDs, the field is not starting at square one, since translational studies on recessive LGMDs provided a framework that can be applied to treating dominant forms of the disease. In this manuscript, we discuss the prospects of treating dominantly inherited forms of LGMD with gene silencing approaches.
Keywords: AAV, dominant myopathy, gene therapy, LGMD, limb-girdle muscular dystrophy, miRNA, RNAi, RNA interference
Current Gene Therapy
Title:RNAi-based Gene Therapy for Dominant Limb Girdle Muscular Dystrophies
Volume: 12 Issue: 4
Author(s): Jian Liu and Scott Q. Harper
Affiliation:
Keywords: AAV, dominant myopathy, gene therapy, LGMD, limb-girdle muscular dystrophy, miRNA, RNAi, RNA interference
Abstract: Limb Girdle Muscular Dystrophy (LGMD) refers to a group of 25 genetic diseases linked by common clinical features, including wasting of muscles supporting the pelvic and shoulder girdles. Cardiac involvement may also occur. Like other muscular dystrophies, LGMDs are currently incurable, but prospective gene replacement therapies targeting recessive forms have shown promise in pre-clinical and clinical studies. In contrast, little attention has been paid to developing gene therapy approaches for dominant forms of LGMD, which would likely benefit from disease gene silencing. Despite the lack of focus to date on developing gene therapies for dominant LGMDs, the field is not starting at square one, since translational studies on recessive LGMDs provided a framework that can be applied to treating dominant forms of the disease. In this manuscript, we discuss the prospects of treating dominantly inherited forms of LGMD with gene silencing approaches.
Export Options
About this article
Cite this article as:
Liu Jian and Harper Q. Scott, RNAi-based Gene Therapy for Dominant Limb Girdle Muscular Dystrophies, Current Gene Therapy 2012; 12 (4) . https://dx.doi.org/10.2174/156652312802083585
DOI https://dx.doi.org/10.2174/156652312802083585 |
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
-
Viral Myocarditis and Dilated Cardiomyopathy: Etiology and Pathogenesis
Current Pharmaceutical Design Animal Models of Diabetes Mellitus: Relevance to Vascular Complications
Current Pharmaceutical Design The Complex Biology of FOXO
Current Drug Targets Diagnostic and Therapeutic Nanoparticles in Cardiovascular Diseases
Current Pharmaceutical Design Hypertension in Type 2 Diabetes Mellitus: Do We Need to Redefine the Role of Sulfonylureas?
Recent Advances in Cardiovascular Drug Discovery (Discontinued) Nitroso-Redox Balance and Modulation of Basal Myocardial Function: An Update from the Italian Society of Cardiovascular Research (SIRC)
Current Drug Targets Peripartum Cardiomyopathy: An Intensivist's Perspective
Current Women`s Health Reviews Homocysteine and Heart Failure: An Overview
Recent Patents on Cardiovascular Drug Discovery Anthracycline Cardiotoxicity: Prevalence, Pathogenesis and Treatment
Current Cardiology Reviews Chaperone Therapy: New Molecular Therapy for Protein Misfolding Diseases with Brain Dysfunction
Current Bioinformatics Smoking and Hypertension: Effects on Clinical, Biochemical and Pathological Variables Due to Isolated or Combined Action on Cardiovascular System
Current Pharmaceutical Design Transthyretin Deposition in Familial Amyloidotic Polyneuropathy
Current Medicinal Chemistry PI3K Pathway Inhibitors: Better Not Left Alone
Current Pharmaceutical Design Zinc, Metallothioneins and Longevity: Interrelationships with Niacin and Selenium
Current Pharmaceutical Design Control of Autoimmune Diseases by the B7-CD28 Family Molecules
Current Pharmaceutical Design Immune Checkpoint Inhibitor Therapy and Risk of Myocarditis: A Review of the Literature
Current Immunology Reviews (Discontinued) The Insular Cortex and Takotsubo Cardiomyopathy
Current Pharmaceutical Design The Role of Anaphylatoxins C3a and C5a in Regulating Innate and Adaptive Immune Responses
Inflammation & Allergy - Drug Targets (Discontinued) Role of Vascular Progenitor Cells in Cardiovascular Disease
Current Pharmaceutical Design Caveolin-3: Its Importance in Muscle Function and Pathology
Current Genomics