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FA-HP-β-CyD is a **folic acid-appended hydroxypropyl-β-cyclodextrin** designed for tumor-targeted therapy. It is a chemically modified cyclodextrin formed by conjugating folic acid to 2-hydroxypropyl-β-cyclodextrin (HP-β-CyD). The addition of folic acid enables selective uptake by cancer cells expressing folate receptors, contributing to tumor cell selectivity. FA-HP-β-CyD induces apoptosis and autophagic cell death (especially through mitophagy) in leukemia cells, primarily in chronic myeloid leukemia (CML) and acute myeloid leukemia (AML) models. Mechanistically, it disrupts cholesterol homeostasis within targeted cells. FA-HP-β-CyD enhances the cytotoxic effects of drugs like imatinib, ponatinib, cytarabine, and venetoclax. Preclinical models demonstrate it is more potent than unconjugated HP-β-CyD, achieving antitumor activity at lower doses and with lower toxicity compared to conventional chemotherapies. FA-HP-β-CyD is under investigation as a novel, tumor-targeted anticancer agent[1][2][3][5].
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