MSI2 inhibitor

MSI2 inhibitors are a class of therapeutic agents designed to target Musashi-2 (MSI2), an RNA-binding protein that regulates the translation of mRNAs involved in cell fate, proliferation, and survival. MSI2 is frequently upregulated in hematological malignancies such as acute myeloid leukemia (AML), chronic myeloid leukemia (CML), and T-cell acute lymphoblastic leukemia (T-ALL), as well as solid tumors like non-small cell lung cancer (NSCLC) and colorectal cancer. By inhibiting MSI2, these compounds disrupt the maintenance of cancer stem cells and can reverse resistance to chemotherapy by blocking the interaction between MSI2 and its target transcripts, such as NUMB and MYC. Several small-molecule inhibitors, including Ro 08-2750 and R12-8-44-3, have been developed for preclinical research to validate MSI2 as a druggable target in oncology.

Other names

Musashi-2 inhibitorMusashi2 inhibitorMusashi 2 inhibitorMSI2 inhibitorsMSI-2 inhibitorsMSI 2 inhibitors