Jin Hyesoo, Noh Wooseong, Kyung Kangwuk, Yeo Woon-Seok, Song Ye Han, Heo Yong-Seok, Kim Dong-Eun
Department of Bioscience and Biotechnology, Konkuk University, Gwangjin-gu, Seoul05029, Republic of Korea.
Department of Chemistry, Konkuk University, Gwangjin-gu, Seoul05029, Republic of Korea.
Bioconjug Chem. 2025 Apr 16;36(4):815-822. doi: 10.1021/acs.bioconjchem.5c00065. Epub 2025 Mar 27.
Acute myeloid leukemia (AML) is a hematologic malignancy characterized by uncontrolled proliferation of abnormal myeloid cells with a generally poor prognosis despite advancements in chemotherapy and stem cell transplantation. To enhance therapeutic efficacy and minimize systemic toxicity, we designed liposomal nanoparticles functionalized with two distinct targeting ligands, a DNA aptamer or fragment-antigen-binding (Fab) antibody, targeting the surface marker transmembrane glycoprotein CD33 antigen (CD33) on AML cells. Aptamer- and Fab-conjugated liposomes (Apt-Lipm and Fab-Lipm, respectively) were prepared and tested for cellular uptake by CD33-positive AML cell lines. Comparative studies revealed that Fab-Lipm exhibited significantly superior binding affinity, targeting efficiency, and cellular uptake compared with Apt-Lipm. Furthermore, we demonstrated the intracellular distribution and endocytic pathways of Fab-Lipm during the cellular uptake. This comparative study of aptamer- and Fab-conjugated liposomes suggests that the Fab-conjugated liposomal system offers enhanced precision in targeting AML cells for the development of effective therapeutic strategies against hematologic malignancies.
急性髓系白血病(AML)是一种血液系统恶性肿瘤,其特征是异常髓系细胞不受控制地增殖,尽管化疗和干细胞移植取得了进展,但总体预后较差。为了提高治疗效果并将全身毒性降至最低,我们设计了用两种不同靶向配体功能化的脂质体纳米颗粒,一种是DNA适配体或片段抗原结合(Fab)抗体,靶向AML细胞表面标志物跨膜糖蛋白CD33抗原(CD33)。制备了分别与适配体和Fab缀合的脂质体(分别为Apt-Lipm和Fab-Lipm),并测试了它们被CD33阳性AML细胞系的细胞摄取情况。比较研究表明,与Apt-Lipm相比,Fab-Lipm表现出显著更高的结合亲和力、靶向效率和细胞摄取。此外,我们展示了Fab-Lipm在细胞摄取过程中的细胞内分布和内吞途径。这项对适配体和Fab缀合脂质体的比较研究表明,Fab缀合脂质体系统在靶向AML细胞方面具有更高的精准性,可用于开发针对血液系统恶性肿瘤的有效治疗策略。