Mechanism of transferrin receptor 1 target and research progress of its targeted therapy in various diseases
10.13200/j.cnki.cjb.004708
- VernacularTitle:转铁蛋白受体1靶点的作用机制及其在多种疾病中靶向治疗的研究进展
- Author:
Hongxin AN
- Publication Type:Journal Article
- Keywords:
Transferrin receptor 1(TfR1);
Iron homeostasis;
Targeted therapy;
Tumors;
Neurodegenerative disease
- From:
Chinese Journal of Biologicals
2026;39(05):626-634+640
- CountryChina
- Language:Chinese
-
Abstract:
Transferrin receptor 1(TfR1), a key membrane protein regulating cellular iron uptake, is extensively involved in physiological processes such as cell proliferation, metabolism, and immune responses. In recent years, advancements in understanding the structure, function, and dysregulated expression mechanisms of TfR1 in diseases have revealed its potential as a therapeutic target in a wide range of pathologies, including cancer, neurodegenerative disorders, and muscular dystrophies. The high expression of TfR1 in numerous tumor types makes it an attractive target for anticancer drug delivery systems. In neurological conditions such as AD, TfR1-mediated transcytosis across the blood-brain barrier(BBB) offers a promising strategy for brain-targeted delivery of biologics such as antibodies and oligonucleotides. For muscular dystrophy treatment, TfR1-targeted antibody-oligonucleotide conjugates(AOCs) have considerably enhanced the tissue specificity and therapeutic efficacy of exon-skipping therapies. Concurrently, the role of TfR1 in cardiac iron metabolism has drawn attention to its potential applications in cardiovascular disease intervention. In this paper, the molecular mechanisms of TfR1 and the research progress of targeted therapy in various diseases are reviewed, focusing on the recent advances in cutting-edge technologies such as drug delivery system, antibody engineering and AOC platform based on TfR1, and discussing the challenges and prospects in clinical transformation, in order to provide new ideas for the precise application and clinical transformation of therapeutic strategies targeting TfR1 in various diseases.
