Paediatrics
Paediatrics

Systematic identification of disease-causing promoter and untranslated region variants in 8040 undiagnosed individuals with rare disease

Genome Med. 2025 Apr 14;17(1):40. doi: 10.1186/s13073-025-01464-2. ABSTRACT BACKGROUND: Both promoters and untranslated regions (UTRs) have critical regulatory roles, yet variants in these regions are …

KF1601, a dual inhibitor of BCR::ABL1 and FLT3, overcomes drug resistance in FLT3+ blast phase chronic myeloid leukemia

Mol Cancer. 2025 Apr 14;24(1):114. doi: 10.1186/s12943-025-02292-z. ABSTRACT Blast phase chronic myeloid leukemia (BP-CML) poses significant clinical challenges due to its drug resistance, resulting from …