SNORD127-targeting antisense oligonucleotides

SNORD127-targeting antisense oligonucleotides (ASOs) are experimental RNA-based therapeutic candidates designed to inhibit the small nucleolar RNA SNORD127. SNORD127 acts as a guide for the 2′-O-methylation (2′-O-Me) of the 18S ribosomal RNA at position G1447 (Gm1447). Research indicates that Gm1447 is significantly upregulated in leukemia stem cells (LSCs) compared to normal hematopoietic cells and is essential for maintaining the high protein synthesis rates required for LSC self-renewal and leukemia progression. By selectively inhibiting SNORD127, these ASOs reduce Gm1447 levels, impairing the clonogenic growth of acute myeloid leukemia (AML) cells while sparing normal hematopoiesis. This approach represents a novel epigenetic strategy for targeting the LSC compartment in AML.

Other names

SNORD127 ASOsSNORD-127 ASOsSNORD 127 ASOsanti-SNORD127 ASOsanti-SNORD-127 ASOsanti-SNORD 127 ASOs