RUNX1 (RUNX Family Transcription Factor 1)

We also highlight broader RUNX/CBFβ inhibitors, alternative therapeutic vulnerabilities linked to fusion-associated cofactors and chromatin regulators, and combination approaches such as BET bromodomain inhibition that enhance antileukemic activity. Together, these findings support the CBFβ-SMMHC-RUNX1 interface as a mechanistically informative and therapeutically actionable target, and illustrate the broader potential of targeting oncogenic transcription factor complexes in core-binding factor AML.

While the patient showed no response to azacitidine/venetoclax, the limited number of cases precludes definitive conclusions about treatment resistance patterns. These findings expand TACC1's oncogenic spectrum to AML and warrant further investigation to determine whether RUNX1::TACC1 represents a clinically distinct molecular subtype and to elucidate its potential therapeutic vulnerabilities.

Greater relapse-associated biological divergence was observed in selected genotype-age strata, particularly in domains related to clonal evolution, treatment resistance, and minimal residual disease. This ALL-focused proof of concept demonstrates the architectural and analytic potential of mechanistic trajectory simulation for hypothesis generation, longitudinal systems modeling, and future integration with real-world longitudinal datasets.

Access to psychological counseling, peer support groups, and genetic counseling can help individuals process their risk, manage anxiety, and make informed decisions. More research is needed to explore effective coping strategies, resilience-building techniques, and patient-centered resources to improve quality of life for those living with a hereditary predisposition to hematologic malignancies.

P2, N=49, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Apr 2026 --> Apr 2027

AKT inhibitors eliminated differences in DNMT3B, HIF1α, and MMP2 expression between normoxia and hypoxia. In hypoxic conditions, CBFβ-MYH11 regulates the AKT/DNMT3B/SHP2 pathway to modulate variations in P53 expression, ultimately hindering the progression of hepatocellular carcinoma.

In conclusion, ceralasertib 160mg BID d1-7 and 15-21 was established as monotherapy dosing with a response rate of 30% in patients with R/R MDS and CMML. NCT03770429.

Our findings establish RUNX1 as a novel druggable TF in EMT-driven CC and propose the identified small molecule for further preclinical validation. Overall, our integrative approach combining single-cell genomics and AI-guided target discovery, and computational drug screening offers a novel framework for identifying therapeutic vulnerabilities in heterogeneous CC.

The results indicate that microRNA-597 directly suppresses RUNX1, while KCNQ1OT1 modulates this interaction. Our approach enabled the simultaneous analysis of dysregulation in three families of transcripts in gastric cancer progression.

The patient was treated using a standard risk ALL protocol and is in remission for two years. This report demonstrates how comprehensive genomic profiling with OGM and long-read sequencing can help resolve atypical findings from conventional methods, enable proper sub-classification and potentially inform risk assessment in relapsed or drug-resistant disease.

Importantly, distinct genetic lesions appear to correlate with specific immunophenotypic patterns, suggesting biologically meaningful disease subsets and providing insights into mechanisms of lineage plasticity and leukemogenesis. This review summarizes the current understanding of the immunophenotypic and genetic landscape of MPAL, emphasizing the integration of morphologic, immunophenotypic, cytogenetic, and molecular findings into diagnosis and subclassification.