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Updated: Mar 16, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
MicroRNAs-424 and 206 are potential prognostic markers in spinal onset amyotrophic lateral sclerosis
Helen M T de Andrade1, Milena de Albuquerque1, Simoni H Avansini2
1Department of Neurology, School of Medicine, University of Campinas - UNICAMP, Campinas, SP, Brazil.
Introduction:
Skeletal muscle microRNAs (miRNAs) are potential candidate biomarkers for amyotrophic lateral sclerosis (ALS) that deserve further investigation.
Objectives:
To identify miRNAs abnormally expressed in the skeletal muscle and plasma of patients with ALS, and to correlate them with parameters of disease progression.
Methods:
Expression profile of miRNAs in muscle was evaluated using an array platform. Subsequently we assessed the plasmatic expression of candidate miRNAs in a set of 39 patients/39 controls. We employed generalized estimating equations to investigate correlations with clinical data.
Results:
We identified 11 miRNAs differentially expressed in the muscle of ALS patients; of these, miR424, miR-214 and miR-206 were validated by qPCR in muscle samples. In plasma, we found only miR-424 and miR 206 to be overexpressed. Baseline expression of miR-424 and 206 correlated with clinical deterioration over time.
Conclusion:
MiR-424 and miR-206 are potential prognostic markers for ALS.

