IPRD funded research by Qing-Xiang (Amy) Sang
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 systemic 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.
Resources
- American Brain Tumor Association
- Boston Children’s Hospital
- National Cancer Institute
- St. Jude Children’s Research Hospital
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