Researcher Qing-Xiang Amy Sang at Florida State University is pioneering a study to develop a reliable model for atypical teratoid/rhabdoid tumor (ATRT), a rare and aggressive childhood brain cancer. Since over 95% of ATRT cases involve the inactivation of the tumor suppressor gene SMARCB1, researchers used CRISPR/Cas9 gene-editing technology to knock out SMARCB1 and TP53 in human induced pluripotent stem cells (Epi-iPSCs). This innovative approach successfully generated an ATRT-like tumor model, maintaining stem cell properties and expressing key ATRT biomarkers at both mRNA and protein levels. Early findings indicate that targeted treatments, including Ribociclib and PTC-209, decreased the viability of these ATRT-like cells, providing a promising foundation for future therapies.

We are proud to partner with Florida State University and some truly amazing and talented individuals to make these research trials possible. We invite you to explore this page to learn about all the incredible, groundbreaking research funded by the Live Like Bella® Pediatric Cancer Research Initiative that will help make pediatric cancer a treatable and curable disease.