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Long intergenic non-protein coding RNA 1257 (LINC01257) is a long non-coding RNA (lncRNA) gene that does not code for any protein but is transcribed as an RNA molecule longer than 200 nucleotides. It is specifically and highly expressed in the t(8;21) subtype of pediatric acute myeloid leukemia, but absent in healthy hematopoietic cells and other AML subtypes. LINC01257's expression promotes leukemia cell proliferation and survival, suggesting an oncogenic role. Therapeutic silencing using siRNA-loaded lipid nanoparticles (LNP-si-LINC01257) reduces AML cell proliferation and viability in vitro, while showing minimal toxicity in healthy cells, supporting its relevance as a highly disease-specific and promising RNA-based therapeutic target. As a lncRNA, LINC01257 is involved in gene regulation, chromatin modification, and possibly signaling pathways that underpin leukemogenesis. There are currently no known clinical drugs targeting LINC01257 beyond experimental siRNA-based approaches. Elevated levels of LINC01257 can serve as a biomarker for diagnosis, prognosis, or stratification of AML patients with the t(8;21) translocation.
RNA interference (RNAi): siRNA delivery leads to sequence-specific silencing of LINC01257 transcript. Silencing LINC01257 impairs AML cell proliferation and survival.
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