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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
Regulation of skeletal muscle development and disease by microRNAs
1Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, TX, 75390, USA, ning.liu@utsouthwestern.edu.
Abstract:
The identification of microRNAs (miRNA) in vertebrates has uncovered new mechanisms regulating skeletal muscle development and disease. miRNAs are inhibitors and act by silencing specific mRNAs or by repressing protein translation. In many cases, miRNAs are involved in physiological or pathological stress, suggesting they function to exacerbate or protect the organism during stress or disease. Although many skeletal muscle diseases differ in clinical and pathological manifestations, they all have a common feature of dysregulation of miRNA expression. In particular, analysis of miRNA expression patterns in skeletal muscle diseases reveals miRNA signatures, showing many miRNAs are dysregulated during disease. Emerging identification of miRNA targets and involvement in genetic regulatory networks serve to reveal new regulatory pathways in skeletal muscle biology. This chapter features the findings pertaining to skeletal muscle miRNAs in skeletal muscle development and disease and highlights therapeutic applications of miRNA-based technology in diagnosis and treatment of skeletal muscle myopathies.
Insights
MicroRNAs (miRNAs) regulate skeletal muscle development and disease by silencing gene expression. Dysregulated miRNA patterns are common in skeletal muscle myopathies, offering potential therapeutic targets.
Area of Science:
- Molecular Biology
- Genetics
- Biochemistry
Background:
- MicroRNAs (miRNAs) are key regulators of gene expression in vertebrates.
- They function by inhibiting mRNA translation or silencing specific mRNAs.
- miRNAs play critical roles in cellular stress responses, influencing disease outcomes.
Purpose of the Study:
- To explore the role of miRNAs in skeletal muscle development and disease.
- To identify miRNA signatures associated with skeletal muscle pathologies.
- To highlight the therapeutic potential of miRNA-based strategies for myopathies.
Main Methods:
- Analysis of miRNA expression patterns in skeletal muscle disease models.
- Identification of miRNA targets and their involvement in regulatory networks.
- Review of current literature on skeletal muscle miRNAs and their functions.
Main Results:
- miRNAs are crucial for regulating skeletal muscle development.
- Dysregulation of miRNA expression is a common hallmark of skeletal muscle diseases.
- Specific miRNA signatures are observed in various myopathies.
- miRNA targets and regulatory networks are being elucidated.
Conclusions:
- miRNAs are vital in skeletal muscle biology, impacting both normal development and disease states.
- Understanding miRNA dysregulation provides insights into disease mechanisms.
- miRNA-based technologies offer promising avenues for diagnosing and treating skeletal muscle myopathies.
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