Progress of biomacromolecule drug nanodelivery systems in the treatment of rare diseases
10.16438/j.0513-4870.2023-1432
- VernacularTitle:生物大分子药物纳米递送系统在罕见病治疗中的研究进展
- Author:
Shu-jie WEI
1
;
Han-xing HE
2
;
Jin-tao HAO
1
;
Qian-qian LV
1
;
Ding-yang LIU
1
;
Shao-kun YANG
1
;
Hui-feng ZHANG
3
;
Chao-xing HE
1
;
Bai XIANG
1
Author Information
1. School of Pharmacy, Hebei Medical University, Shijiazhuang 050017, China
2. Department of Urology, Shijiazhuang People′s Hospital, Shijiazhuang 050011, China
3. Department of Pediatrics, The Second Hospital of Hebei Medical University, Shijiazhuang 050000, China
- Publication Type:Research Article
- Keywords:
rare disease;
nanocarrier;
biomacromolecule drug;
efficient delivery;
treatment
- From:
Acta Pharmaceutica Sinica
2024;59(7):1952-1961
- CountryChina
- Language:Chinese
-
Abstract:
Rare diseases still lack effective treatments, and the development of drugs for rare diseases (known as orphan drugs) is an urgent medical problem. As natural active ingredients in living organisms, some biomacromolecule drugs have good biocompatibility, low immunogenicity, and high targeting. They have become one of the most promising fields in drug research and development in the 21st century. However, there are still many obstacles in terms of in vivo delivery. In view of the unique advantages of nanocarriers prepared from polymers, lipids, organic biomimetic and inorganic materials in drug delivery, researchers are committed to building an efficient delivery system with versatility and synergy to solve the bottleneck issues in treating rare diseases with biomacromolecule drugs. Therefore, this article reviews the research progress of nanocarrier delivering proteins, peptides and nucleic acids in the field of rare disease treatment in the past ten years, which provides ideas for researches on biomacromolecule drug nanosystems in the field of treatment of rare diseases.
