Targeting an RNA Methyltransferase to Treat Neuroblastoma

Grant awarded in 2024 following an in-depth review of high-quality funding applications by our world class Scientific Advisory Board.

Associate Professor Tao Liu from the Children’s Cancer Institute proposal researched the potential to improve patient outcomes from neuroblastoma treatments by targeting an RNA Methyltransferase.

Targeting an RNA Methyltransferase to Treat Neuroblastoma

Despite the availability of multimodality therapies, neuroblastoma still accounts for approximately 15% of all childhood cancer deaths.

Due to the cytotoxic side effects of current chemo-radiotherapies on normal tissues, survivors often experience long-term health issues, including infertility.

Assoc. Prof. Liu and his team have recently developed a new pipeline to identify neuroblastoma drivers and their inhibitors.

This pipeline integrates RNA-sequencing data from the neuroblastoma tissues of 493 patients with genome-wide survival analyses of every gene. The result is a short list of overexpressed transcripts that are significantly and independently correlated with poor prognosis.

Notably, Assoc. Prof. Liu has already found that an RNA methyltransferase known as BUD23 is one of the top transcripts highly expressed in neuroblastomas and independently associated with poor patient outcomes.

In this project, Assoc. Prof. Liu and his team aim to demonstrate the oncogenic role of the RNA methyltransferase in neuroblastoma, develop its inhibitors, and showcase the significant anticancer effects of these inhibitors, paving the way for their potential translation into safer and more effective treatments for neuroblastoma.

We would like to express our thanks to the MANILDRA FOUNDATION for supporting this research grant.

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