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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
MicroRNAs: a light into the "black box" of neuropediatric diseases?
Ahmed Omran1, Dalia Elimam, Sherien Shalaby
1Department of Pediatrics, Xiangya Hospital of Central South University, No. 87 Xiangya Road, Changsha, 410008, Hunan, China.
Insights
MicroRNAs (miRNAs) are crucial in pediatric neurological conditions. Understanding their role offers new therapeutic strategies for treating childhood brain diseases and preventing long-term disabilities.
Area of Science:
- Neurology
- Molecular Biology
- Genetics
Background:
- Pediatric central nervous system (CNS) diseases cause significant lifelong impairments and burdens.
- Despite advances, the exact causes and pathogenesis of these disorders remain unclear.
- MicroRNAs (miRNAs) are increasingly recognized for their roles in CNS development and neuropsychiatric diseases.
Purpose of the Study:
- To review the involvement of miRNAs in various pediatric neurological conditions.
- To highlight the potential of miRNA-based research for novel therapeutic development.
- To emphasize the importance of understanding miRNAs for preventing pediatric disabilities.
Main Methods:
- Literature review of recent studies on miRNAs and pediatric neurological disorders.
- Analysis of the role of miRNAs in CNS development and disease pathogenesis.
- Synthesis of current knowledge on miRNA-based therapeutic strategies.
Main Results:
- MicroRNAs play a significant role in the development and progression of pediatric neurological diseases.
- Specific miRNAs are implicated in various conditions affecting the pediatric CNS.
- Targeting miRNAs presents a promising avenue for future treatments.
Conclusions:
- MicroRNA research is vital for understanding and combating pediatric neurological disorders.
- MiRNA-based therapies hold the potential to improve outcomes and prevent disabilities.
- Further investigation into miRNAs is essential for advancing pediatric neurology.
Abstract:
Although there have been tremendous advances in the diagnosis and treatment of pediatric brain diseases in the last few decades, the causes and pathogenesis of these diseases remain to be elucidated. Pediatric central nervous system (CNS) diseases create both short- and long-term impairments and disabilities and are therefore one of the leading causes for emotional, financial, and social burden to patients, their families, and their social network. The significant pediatric morbidity and mortality rates caused by CNS diseases call for equally significant efforts toward a better understanding of the etiology and nature of these disorders. Recent studies show the involvement of microRNAs (miRNAs) in various aspects of central nervous system development and neuropsychiatric diseases. This review focuses on the role of miRNAs in different pediatric neurological conditions. We emphasize the importance of microRNA-based research in combating pediatric neurological disorders. We believe this approach will result in novel therapies to secure normal development and prevent disabilities in the pediatric population.
