RARE DISEASE RESEARCH & TRAINING
IPRD funded research by Qing-Xiang (Amy) Sang, Ph.D.
Atypical Teratoid Rhabdoid Tumor(ATRT) is rare pediatric brain tumor that is highly heterogeneous, invasive, and usually fatal. Current treatments for ATRT include surgery, radiotherapy, and chemotherapy, which entail high risks of neurocognitive impairment and toxicities. Moreover, considerable molecular heterogeneity across ATRT subgroups potentially lowers treatment efficacy and complicates the development of novel treatment strategies. The ATRT subgroups are characterized by unique epigenomic and transcriptomic profiles. Subgroup-specific therapy development represents a major advancement toward achieving therapeutic precision and enhanced treatment outcomes. This research project will design and develop siRNAs (small interfering RNAs) platforms to silence subgroup-specific genes such as PTCH1 and GLI2 in ATRT-SHH and VEGFA as candidate therapeutic siRNAs.
More Information onAtypical Teratoid Rhabdoid Tumor (ATRT) & Diffuse Intrinsic Pontine Glioma (DIPG) Research
An IPRD-funded research team led by Qing-Xiang Amy Sang, Ph.D., in FSU’s Department of Chemistry and Biochemistry is advancing new therapies for rare pediatric brain tumors, including ATRT and DIPG.
Sang’s lab is exploring gene expression patterns and tumor microenvironment interactions to uncover promising treatment strategies, with research contributions from multiple graduate and student researchers, including Faiza Mahmud, Uvindu Thilanka Dasili Wickramasinghe, Jonathan Jocelyn, Marian Gonzalez, Sophia Martinez, and Fiona Dininger.