ML-243 is primarily used in biochemical research to study cellular processes, particularly in the context of cancer and neurodegenerative diseases. It acts as a selective inhibitor of the deubiquitinating enzyme USP1, which plays a role in DNA repair and cell cycle regulation. By inhibiting USP1, this compound helps researchers understand the mechanisms of DNA damage response and its implications in cancer therapy. Additionally, it is utilized in drug discovery efforts to identify potential therapeutic agents targeting USP1-related pathways. Its application extends to investigating the role of ubiquitination in protein degradation and cellular signaling, providing insights into disease mechanisms and potential treatment strategies.