anti-cPCMTD1 gapmer

Anti-cPCMTD1 gapmer is a locked nucleic acid (LNA)-modified, ribonuclease H (RNase H)-recruiting antisense oligonucleotide (gapmer) designed to specifically target and degrade the circular RNA PCMTD1 (circPCMTD1) without affecting the linear PCMTD1 transcript. Developed by researchers at New York University School of Medicine and collaborating institutions, the gapmer is formulated in lipid nanoparticles (LNPs) for in vivo delivery. circPCMTD1 is a protein-coding circular RNA that is overexpressed in advanced-stage chronic myeloid leukemia (CML), particularly in blast crisis (CML-BC). It encodes a 127-amino-acid peptide (cPCMTD1-127aa) that interacts with the BCR/ABL1 oncoprotein and components of the BTR complex (BLM, TOP3A, and RMI1) to resolve replication stress and promote cell survival. By knocking down circPCMTD1, the gapmer disrupts BTR complex formation, activates the DNA damage response pathway, induces G2/M cell cycle blockade, and inhibits the proliferation of BCR/ABL1-positive leukemic cells.

Other names

anti-circPCMTD1 gapmeranti-circPCMTD-1 gapmeranti-circPCMTD 1 gapmercircPCMTD1-targeting gapmercircPCMTD-1-targeting gapmercircPCMTD 1-targeting gapmer