Surprisingly, we found that 11q deletions are enriched in neuroblastomas with ATRX deletions compared to a reference cohort, but not in neuroblastomas with ATRX point mutations. Taken together our data emphasizes a distinct ATRX mutation spectrum in neuroblastoma, which should be considered when studying molecular phenotypes and therapeutic strategies.
To generate the first primary mouse model of soft tissue sarcoma, 4-hydroxytamoxifen (4-OHT) was injected into the gastrocnemius muscle... Collectively, these results show that loss of ATRX function impairs the cGAS-STING signaling pathway in soft tissue sarcomas and promotes their response to radiation therapy and oncolytic virus therapy. These findings identify ATRX as a biomarker in treatment response and identify novel genomically-informed therapeutic strategies for treatment of soft tissue sarcoma.
Based on IHC and genetic analyses of the biopsied samples, three patients were enrolled into the Phase 0/2 clinical trial and baseline value were used for PD analysis. CONCLUSION Single-pass stereotactic needle biopsy is a feasible and safe strategy to collect pre-treatment tissue in support of a Phase 0 clinical trial, enabling CLIA-certified genetic analysis for trial screening and tumor PD analysis.
DMG-H3 tend to develop in adolescence with a similar dismal prognosis like GBM and HAP in the elderly. We here describe spinal HAP with a distinct molecular profile for the first time.