Background: Neuroblastoma is the most common extracranial solid tumor in infancy.
The majority of children have a disseminated disease at diagnosis with bone marrow as
the most common site of metastasis. Although several prognostic factors have been defined
(i.e. age, stage, histology, recurrent genetic anomalies), the identification of non-invasive
biomarkers for disease follow-up and therapy monitoring is indeed still a clinical need. Aberrant
regulation of microRNAs (miRNAs) expression has been implicated in several malignancies.
Objectives: In this mini-review, we describe the recent findings about miRNAs in neuroblastoma,
both in the tumor and circulation, with particular focus on those involved in tumor progression
and drug resistance. Furthermore, we will discuss the use of specific miRNAs as potential
therapeutic tools in this tumor.
Results: Several miRNAs have been identified to be down- or up-regulated in primary tumors
and have been associated with MYCN amplification, differentiation, dissemination and
chemoresistance. Little evidence is available in the literature about circulating miRNAs which
are of particular interest as potential biomarkers for liquid biopsy.
Conclusion: Identification of body-fluid markers for non-invasive diagnosis, risk stratification,
treatment monitoring and tumor follow-up, is gaining growing interest, especially in the
pediatric field. miRNAs are suitable candidates as biomarkers in neuroblastoma but further
investigations are needed to expand knowledge regarding their role in this malignancy to design
specific approaches of miRNAs-mediated therapies.