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Liposomal bortezomib is a nanoparticle drug delivery formulation of the small-molecule proteasome inhibitor bortezomib. By encapsulating bortezomib within liposomes, the formulation aims to overcome the drug's poor pharmacokinetic profile, extend its systemic circulation time, and reduce off-target toxicities, most notably peripheral neuropathy. The active moiety, bortezomib, reversibly inhibits the 26S proteasome, leading to cell cycle arrest and apoptosis in malignant cells. Research primarily focuses on its application in hematologic malignancies such as multiple myeloma and acute myeloid leukemia (AML), as well as solid tumors like neuroblastoma. Preclinical studies have demonstrated that liposomal encapsulation can significantly enhance therapeutic efficacy and survival compared to free bortezomib, particularly in models of AML with specific genetic defects like MLL-PTD and FLT3-ITD.
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