anti-miR-27 antisense oligonucleotide

anti-miR-27 antisense oligonucleotide is a therapeutic candidate being developed for the treatment of Parkinson's disease. It consists of an antisense oligonucleotide (ASO) designed to target and inhibit the miR-27 family, which includes miR-27a and miR-27b. These microRNAs are highly expressed in the substantia nigra and thalamus and have been found to be upregulated in patients with Parkinson's disease. miR-27 is known to regulate key pathways involved in the disease's pathogenesis, including mitochondrial dysfunction, inflammation, and oxidative stress. By inhibiting miR-27, the ASO aims to de-repress target mRNAs and restore cellular function, potentially offering a disease-modifying effect. Preclinical data has shown over 95% knockdown of miR-27 in vivo following intracranial administration and successful target engagement in human iPSC-derived neurons.

Other names

anti-miR-27 ASOanti-miR27 ASOanti-miR 27 ASOmiR-27 antisense oligonucleotidemiR27 antisense oligonucleotidemiR 27 antisense oligonucleotideanti-miR-27anti-miR27anti-miR 27miR-27 inhibitormiR27 inhibitormiR 27 inhibitor