Clofarabine-loaded aptamer-conjugated biodegradable nanoparticle successfully targeted CD117 overexpressed HL60 cells and potentially induced apoptosis.

Acute Myeloid Leukemia (AML) is a rapidly progressing malignancy characterized by the proliferation of abnormal neutrophils, leading to severe symptoms and complications. Current widely used treatment options include chemotherapy and radiotherapy, which often result in suffering from systemic toxicity and drug resistance. To mitigate systemic toxicity and off-target side effects, a targeted therapeutic strategy is one of the remarkably successful options. For targeting AML cells, we have chosen a single-strand DNA aptamer (Apt), which is specific for the biomarker CD117, overexpressing AML cells. This study introduces explicitly a novel therapeutic approach employing aptamer-conjugated clofarabine-loaded PLGA nanoparticles (Apt-CNP) targeting the CD117 receptor on HL60 leukemia cells. Clofarabine, a potent nucleoside analogue, disrupts DNA synthesis and induces cancer cell death but is limited by its toxicity and resistance. Encapsulation in PLGA nanoparticles enables sustained drug release, maintaining therapeutic concentrations and potentially reducing drug resistance. Our findings demonstrate that Apt-CNP effectively targets HL60 leukemia cells, thereby improving drug delivery and reducing adverse effects on healthy cells. This targeted approach may open a new avenue for more specific drug delivery to mobile and floated blood cells, including AML (HL60 leukemia) cells, and overcome the limitations of traditional AML treatments.