ASO-miR-29b

ASO-miR-29b is an **antisense oligonucleotide** designed to sterically block the miR-29b binding site in the 3′ untranslated region (3′ UTR) of the human *GRN* (progranulin) mRNA. This selective blockade prevents miR-29b from repressing translation, thereby upregulating endogenous progranulin protein levels. The main therapeutic aim of ASO-miR-29b is to address progranulin haploinsufficiency caused by heterozygous *GRN* mutations, such as those observed in **frontotemporal dementia (FTD)**. The ASO increases progranulin in cultured cells, iPSC-derived neurons, and mouse brain by displacing miR-29b from its mRNA target, which derepresses progranulin synthesis[1][2][6]. The ASO is engineered for CNS stability and specificity (e.g., 2′-O-methoxyethyl ribose, phosphorothioate backbone) and acts through a competitive, direct blocking mechanism at the miRNA-mRNA interface. Its use is agnostic to specific *GRN* mutations, targeting the underlying mechanism of reduced protein translation in FTD[1][2][6].

Other names

ASO targeting miR-29b binding siteantisense oligonucleotide targeting miR-29b site