MicroRNA-27b (miR-27b)

MicroRNA-27b (miR-27b) is a highly conserved non-coding RNA that functions as a post-transcriptional regulator of gene expression by binding to the 3' untranslated regions (UTRs) of specific messenger RNAs. In the context of neurodegenerative disease, miR-27b is significantly upregulated in patients with Parkinson's disease, where it plays a pathological role by suppressing the expression of PTEN-induced kinase 1 (PINK1). PINK1 is essential for the process of mitophagy, the selective degradation of damaged mitochondria; its suppression by miR-27b leads to mitochondrial dysfunction, increased oxidative stress, and the eventual death of dopaminergic neurons. The therapeutic candidate NMT.002 is an antisense oligonucleotide (ASO) designed to bind and inhibit miR-27b, thereby de-repressing PINK1 and restoring mitochondrial health. Beyond its role in the central nervous system, miR-27b is involved in regulating angiogenesis, adipogenesis, and various oncogenic pathways, acting as either a tumor suppressor or an oncomir depending on the tissue context.

Other names

MIR27Bhsa-miR-27bmicroRNA 27bmiR-27b-3phsa-mir-27b

NMT.002 is an antisense oligonucleotide (ASO) that acts as an antagomir by binding with high affinity to the sequence of MicroRNA-27b. This binding prevents miR-27b from interacting with its target mRNAs, such as PINK1, thereby reversing the microRNA-mediated translational repression and promoting neuroprotective mitochondrial clearance (mitophagy).