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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
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Current Status of microRNA-Based Therapeutic Approaches in Neurodegenerative Disorders

Sujay Paul1, Luis Alberto Bravo Vázquez1, Samantha Pérez Uribe1

  • 1Tecnologico de Monterrey, School of Engineering and Sciences, Campus Queretaro, Av. Epigmenio Gonzalez, No. 500 Fracc. San Pablo, Querétaro CP 76130, Mexico.

Cells
|July 19, 2020
PubMed

Insights

MicroRNAs (miRNAs) are crucial gene regulators implicated in neurodegenerative disorders (NDDs). This review explores miRNA-based therapeutics for NDDs like Alzheimer's and Parkinson's, discussing delivery and potential.

Area of Science:

  • Biochemistry and Molecular Biology
  • Genetics and Genomics
  • Neuroscience

Background:

  • MicroRNAs (miRNAs) are vital gene regulators involved in numerous biological and pathological processes.
  • Growing evidence links specific miRNAs to the pathogenesis of neurodegenerative disorders (NDDs).

Purpose of the Study:

  • To review current research on miRNA-based therapeutics for prevalent and lethal NDDs.
  • To discuss delivery methods, outcomes, and limitations of miRNA therapeutics.

Main Methods:

  • Comprehensive literature review of studies on miRNA therapeutics for NDDs.
  • Analysis of miRNA roles in Alzheimer's, Parkinson's, Huntington's, ALS, Friedreich's Ataxia, SMA, and FTD.

Main Results:

  • Identified specific miRNAs implicated in the progression of various NDDs.
  • Evaluated current strategies for delivering miRNA therapeutics to the central nervous system.
  • Assessed the therapeutic potential and challenges associated with miRNA-based treatments.

Conclusions:

  • miRNA-based therapeutics show significant promise for treating neurodegenerative diseases.
  • Further research into delivery systems and clinical efficacy is essential for realizing their full potential.
Keywords:
MicroRNAmiRNA deliverymiRNA therapeuticsneurodegenerative disorders

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