Researchers have discovered a method to transform cells from a deadly and aggressive type of pediatric cancer into muscle cells. Professor Christopher Vakoc and CSHL School of Biological Sciences graduate Martyna Sroka discovered a way to transform rhabdomyosarcoma cells into muscle cells. Rhabdomyosarcoma (RMS) is a devastating and aggressive type of pediatric cancer. Nobody knew if this proposed treatment method, known as differentiation therapy, would ever work in RMS. But, thanks to Vakoc’s lab, it now appears to be a real possibility. Vakoc and his team developed a new genetic screening technique to carry out their mission. They used genome-editing technology (CRISPR) to find genes that, when disrupted, force RMS cells to become muscle cells. That’s when the NF-Y protein appeared. The scientists witnessed an astonishing transformation when NF-Y was impaired.
Previously, Vakoc and his team succeeded in transforming Ewing sarcoma cells into healthy tissue cells. The Ewing sarcoma and RMS discoveries were supported by local families who’d lost loved ones to these cancers.
Reference: Sroka MW, Skopelitis D, Vermunt MW, et al. Myo-differentiation reporter screen reveals NF-Y as an activator of PAX3–FOXO1 in rhabdomyosarcoma. Proc Natl Acad Sci. 2023;120(36):e2303859120. doi: 10.1073/pnas.2303859120