Long intergenic non-protein coding RNA 1257 (LINC01257) (LINC01257)

Long intergenic non-protein coding RNA 1257 (LINC01257) is a long non-coding RNA (lncRNA) that acts as a specific oncogenic driver in pediatric acute myeloid leukemia (AML) (Connerty et al., 2021). It is uniquely characterized by its high expression in AML patients harboring the t(8;21) chromosomal translocation, while being virtually undetectable in healthy bone marrow and peripheral blood cells (Connerty et al., 2021). In these leukemia cells, LINC01257 is essential for maintaining cell growth and survival; its knockdown leads to a significant reduction in proliferation and the induction of apoptosis (Connerty et al., 2021). Due to its highly restricted expression pattern, LINC01257 represents an attractive therapeutic target for precision medicine, offering a way to selectively eliminate cancer cells while sparing healthy tissue (Connerty et al., 2021). Experimental therapeutic strategies involve the use of siRNA-loaded lipid nanoparticles (LNPs) to achieve targeted silencing of LINC01257, which has shown efficacy in preclinical models (Connerty et al., 2021). This approach leverages the same lipid nanoparticle technology used in FDA-approved RNAi therapies like Patisiran (Connerty et al., 2021).

Other names

LINC01257Long non-coding RNA LINC01257Long intergenic non-protein coding RNA 1257ENSG00000258636t(8;21)-specific lncRNA

RNA interference (RNAi) mediated silencing using siRNA delivered via lipid nanoparticles (LNPs) to inhibit oncogenic signaling and induce apoptosis in leukemia cells.