ML216

ML216 is a potent and selective small molecule inhibitor of the Bloom syndrome protein (BLM) helicase, a member of the RecQ family of DNA helicases. BLM is a crucial enzyme involved in maintaining genomic stability by resolving DNA structures such as G-quadruplexes and Holliday junctions during replication and repair. ML216 specifically inhibits the DNA unwinding activity of BLM, which leads to an increase in sister chromatid exchanges and sensitizes cells to DNA-damaging agents like cisplatin, melphalan, and ionizing radiation. Beyond its role as a chemical probe in DNA repair research, ML216 has demonstrated preclinical efficacy in various cancer models, including prostate cancer, multiple myeloma, and colorectal cancer with microsatellite instability (MSI). Additionally, recent studies have explored its potential in treating age-related cardiac fibrosis and pulmonary fibrosis by modulating TGF-β1 signaling and preventing DNA damage-induced cellular senescence.

Other names

Bloom syndrome protein inhibitorBLM helicase inhibitor