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RNA binding motif protein, X-linked-like 1 (RBMXL1) is a protein-coding gene that encodes an RNA binding protein highly homologous to RBMX. RBMXL1 is the only known functional retrogene of RBMX, maintaining an intact open reading frame and expressed in multiple tissues. RBMXL1 is involved in post-transcriptional RNA processing, particularly alternative splicing and transcriptional regulation of heterochromatin. In hematological malignancies such as acute myeloid leukemia (AML), RBMXL1 is consistently overexpressed and, together with RBMX, is necessary for leukemia cell survival. Mechanistically, RBMXL1 binds to target mRNAs (notably CBX5) and modulates chromatin compaction and genome integrity. Loss of RBMXL1 impairs chromatin architecture, leading to increased genome instability and altered leukemia cell proliferation and apoptosis. Therefore, RBMXL1 represents a potential novel therapeutic target and biomarker in AML, though no approved drugs are known to directly modulate its function[1][3].
Not established for direct drug targeting; potential mechanisms include disruption of RNA binding, inhibition of chromatin regulation pathways
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