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An anti-miR-21 inhibitor is a therapeutic agent designed to target and inhibit microRNA-21 (miR-21), an oncogenic and pro-fibrotic microRNA that is highly upregulated in various cancers, cardiovascular diseases, and fibrotic disorders. miR-21 plays a critical role in regulating cell proliferation, apoptosis, migration, and tissue remodeling by suppressing tumor suppressor genes and other regulatory proteins such as PTEN, PDCD4, and Sprouty 1/2. Inhibition of miR-21 can be achieved through various modalities, most notably antisense oligonucleotides (such as antagomirs or locked nucleic acids) that bind and sequester mature miR-21, as well as small molecules that disrupt pre-miR-21 processing by Dicer. In preclinical and clinical settings, anti-miR-21 inhibitors (such as lademirsen) have been investigated for the treatment of Alport syndrome, diabetic nephropathy, and various malignancies, demonstrating potential to reduce fibrosis, restore tumor suppressor expression, and sensitize cancer cells to chemotherapy.
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