MALAT1 gapmer

MALAT1 gapmer refers to a class of gapmer antisense oligonucleotides (ASOs) designed to target and degrade the long non-coding RNA (lncRNA) Metastasis-Associated Lung Adenocarcinoma Transcript 1 (MALAT1). These gapmers typically consist of a central DNA core flanked by chemically modified RNA-like segments (such as locked nucleic acids [LNA] or 2'-O-methoxyethyl [2'-MOE] modifications) that enhance binding affinity and nuclease resistance. Upon hybridization to MALAT1, the DNA/RNA duplex recruits RNase H, which cleaves and degrades the target lncRNA. MALAT1 is highly conserved and overexpressed in various cancers (such as triple-negative breast cancer and multiple myeloma) and fibrotic diseases, where it regulates gene expression, alternative splicing, and cell-cell interactions. Preclinical development of MALAT1 gapmers has been pursued by companies like Ionis Pharmaceuticals and Flamingo Therapeutics (with candidates like FTX-001 / FLM-7523), as well as academic institutions investigating its role in oncology and kidney fibrosis.

Other names

MALAT1 ASOMALAT-1 ASOMALAT 1 ASOMALAT1 antisense oligonucleotideMALAT-1 antisense oligonucleotideMALAT 1 antisense oligonucleotideMALAT1 gapmer ASOMALAT-1 gapmer ASOMALAT 1 gapmer ASOgapmer-MALAT1gapmer-MALAT-1gapmer-MALAT 1