NIH R01 · 2025
Genomic correlates of outcome in pleuropulmonary blastoma
Project Summary/Abstract Pleuropulmonary blastoma (PPB) is the most common pediatric lung cancer in children and one of the sentinel manifestations of DICER1-related tumor predisposition. While some patients with PPB may be treated with surgery alone, children with advanced PPB require an intense combination of surgery, chemotherapy and often radiation. Unresectable or recurrent disease is associated with a poor prognosis. Currently, there are no molecular factors sufficiently predictive to allow risk stratification in advanced PPB and no ways to rationally target oncogenic DICER1 variants. The DICER1 enzyme normally produces mature microRNAs by cleaving the two arms of a pre-microRNA…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.