Neuroblastoma: Emerging trends in pathogenesis, diagnosis, and therapeutic targets

Santharam S. Katta, Veerababu Nagati, Atreya S.V. Paturi, Swati P. Murakonda, Ajay B. Murakonda, Manoj K. Pandey, Subash C. Gupta, Anil K. Pasupulati, Kishore B. Challagundla

Research output: Contribution to journalArticlepeer-review

4 Scopus citations


Neuroblastoma (NB) accounts for about 13% of all pediatric cancer mortality and is the leading cause of pediatric cancer death for children aged 1 to 5 years. NB, a developmental malignancy of neural ganglia, originates from neural crest-derived cells, which undergo a defective sympathetic neuronal differentiation due to genomic and epigenetic aberrations. NB is a complex disease with remarkable biological and genetic variation and clinical heterogeneity, such as spontaneous regression, treatment resistance, and poor survival rates. Depending on its severity, NB is categorized as high-risk, intermediate-risk, and low-risk., whereas high-risk NB accounts for a high infant mortality rate. Several studies revealed that NB cells suppress immune cell activity through diverse signaling pathways, including exosome-based signaling pathways. Exosome signaling has been shown to modulate gene expression in the target immune cells and attenuate the signaling events through non-coding RNAs. Since high-risk NB is characterized by a low survival rate and high clinical heterogeneity with current intensive therapies, it is crucial to unravel the molecular events of pathogenesis and develop novel therapeutic targets in high-risk, relapsed, or recurrent tumors in NB to improve patient survival. This article discusses etiology, pathophysiology, risk assessment, molecular cytogenetics, and the contribution of extracellular vesicles, non-coding RNAs, and cancer stem cells in the tumorigenesis of NB. We also detail the latest developments in NB immunotherapy and nanoparticle-mediated drug delivery treatment options.

Original languageEnglish (US)
Pages (from-to)444-459
Number of pages16
JournalJournal of Controlled Release
StatePublished - May 2023

All Science Journal Classification (ASJC) codes

  • Pharmaceutical Science


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