Developing an accurate genetic model for rhabdomyosarcoma therapy

In simple terms, what is your project about?
Developing a new mouse model for rhabdomyosarcoma therapy testing.

What type of childhood cancer does your project focus on?
Rhabdomyosarcoma (RMS) is the most common pediatric soft-tissue sarcoma. Fortunately, through modern medical advances most children with localized tumors can be cured. However, children that present with metastatic disease at the time of diagnosis have a poor prognosis and survival rate.

What do you hope to achieve through your research and what will it mean to the world of childhood cancer if your project is successful?
Our long-term goal is to understand the role of alternative splicing in cancer and to target the splicing pathway as a therapeutic intervention point in pediatric RMS.  We hope to find cures for those patients with metastasis that are not responsive to the current treatment regimens.

In simple terms, how you will conduct your research? (For example,  is it lab based, is it a clinical trial, are children directly involved?)
We are working in a laboratory setting to generate an accurate mouse model for RMS. We will use this model to test novel treatments.

What is innovative about your approach?
There are currently no mouse models for RMS that account for the heterogeneous genetic signatures characteristic of the RMS disease in patients.