Abstract 5423: Novel targeted therapy for neuroblastoma: Silencing the MXD3 gene using siRNA

Neuroblastoma is a cancer of the sympathetic nervous system and the most common extracranial solid tumor in children. Almost half of neuroblastoma patients have a high-risk phenotype at diagnosis: metastatic disease. Prognosis of these patients is very poor with only 30% survival despite the most intensive therapies currently available. In addition, patients who respond successfully to treatment suffer from side effects from chemo and radiation therapies, leading to life-long irreversible complications. Therefore, there is a desperate need for a neuroblastoma-targeted therapy that is more effective and has fewer side effects than conventional therapies. Our goal is to develop a molecular-targeted therapy that can be used in tandem with current treatments. In our previous study, we identified the transcription factor MXD3 as a novel molecular target for neuroblastoma (presented in the 2013 AACR annual meeting). In the current study, we demonstrated in vitro efficacy of an MXD3 targeted therapeutic using siRNA and nanoparticles. We have developed MXD3 targeting methods using MXD3 siRNA and superparamagnetic iron oxide nanoparticles (NPs). We assembled siRNA on the surface of NPs and introduced them to SK-N-DZ cells (neuroblastoma cell line with high MXD3 expression) in vitro. Upon a single treatment with the siRNA-NP-complexes, the siRNAs were successfully delivered into the cells and MXD3 knockdown was confirmed at the protein level as early as 4 hours post treatment. The cell...
Source: Cancer Research - Category: Cancer & Oncology Authors: Tags: Clinical Research (Excluding Clinical Trials) Source Type: research