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

Progress of biomacromolecule drug nanodelivery systems in the treatment of rare diseases

目前罕见病仍缺乏有效的治疗方法,罕见病药物(称孤儿药)的研发是亟需解决的医学难题。一些生物大分子药物作为生物体内的天然活性成分,具有良好的生物相容性、低免疫原性、高靶向性,已成为21世纪药物研发中极具前景的领域之一,但在体内递送方面仍存在诸多障碍,鉴于由聚合物、脂质、有机仿生和无机材料等制备的纳米载体在药物递送方面的独特优势,研究者致力于构建多功能性与协同作用的高效递送系统,以解决罕见病治疗中生物大分子药物目前存在的瓶颈问题。因此,本文对近10年来纳米载体递送蛋白、多肽及核酸等药物在罕见病治疗领域的研究进展进行系统综述,为基于生物大分子药物纳米递送系统的罕见病干预研究提供思路。

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.