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Progress of biomacromolecule drug nanodelivery systems in the treatment of rare diseases
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Shu-jie WEI1, Han-xing HE2, Jin-tao HAO1, Qian-qian LV1, Ding-yang LIU1, Shao-kun YANG1, Hui-feng ZHANG3, Chao-xing HE1, *, Bai XIANG1, *
Acta Pharmaceutica Sinica | 2024, 59(7) : 1952 - 1961
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Acta Pharmaceutica Sinica | 2024, 59(7): 1952-1961
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Progress of biomacromolecule drug nanodelivery systems in the treatment of rare diseases
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Shu-jie WEI1, Han-xing HE2, Jin-tao HAO1, Qian-qian LV1, Ding-yang LIU1, Shao-kun YANG1, Hui-feng ZHANG3, Chao-xing HE1, *, Bai XIANG1, *
Affiliations
  • 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
Published: 2024-07-12 doi: 10.16438/j.0513-4870.2023-1432
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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.

rare disease  /  nanocarrier  /  biomacromolecule drug  /  efficient delivery  /  treatment
Shu-jie WEI, Han-xing HE, Jin-tao HAO, Qian-qian LV, Ding-yang LIU, Shao-kun YANG, Hui-feng ZHANG, Chao-xing HE, Bai XIANG. Progress of biomacromolecule drug nanodelivery systems in the treatment of rare diseases[J]. Acta Pharmaceutica Sinica, 2024 , 59 (7) : 1952 -1961 . DOI: 10.16438/j.0513-4870.2023-1432
Year 2024 volume 59 Issue 7
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doi: 10.16438/j.0513-4870.2023-1432
  • Receive Date:2023-12-22
  • Online Date:2025-11-26
  • Published:2024-07-12
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  • Received:2023-12-22
  • Revised:2024-05-16
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Affiliations
    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
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表12种不同金属材料的力学参数

Family
属数
Number of
genus
种数
Number of
species
占总种数比例
Percentage of
total species (%)

Genus
种数
Number of
species
占总种数比例
Percentage of total
species (%)
鹅膏菌科Amanitaceae 2 11 5.26 鹅膏菌属 Amanita 10 4.78
小菇科 Mycenaceae 2 12 5.74 丝盖伞属 Inocybe 5 2.39
多孔菌科 Polyporaceae 8 14 6.70 蜡蘑属 Laccaria 5 2.39
红菇科 Russulaceae 3 23 11.00 小皮伞属 Marasmius 6 2.87
小菇属 Mycena 11 5.26
光柄菇属 Pluteus 5 2.39
红菇属 Russula 17 8.13
栓菌属 Trametes 5 2.39
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