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聚焦遗传性肾脏疾病:呼吁药物传递和纳米医学解决方案。

Spotlight on Genetic Kidney Diseases: A Call for Drug Delivery and Nanomedicine Solutions.

机构信息

Department of Biomedical Engineering, University of Southern California, Los Angeles, California 90089, United States.

John A. Paulson School of Engineering & Applied Sciences, Harvard University, Allston, Massachusetts 02134, United States.

出版信息

ACS Nano. 2023 Apr 11;17(7):6165-6177. doi: 10.1021/acsnano.2c12140. Epub 2023 Mar 29.

Abstract

Nanoparticles as drug delivery carriers have benefited diseases, including cancer, since the 1990s, and more recently, their promise to quickly and efficiently be mobilized to fight against global diseases such as in the COVID-19 pandemic have been proven. Despite these success stories, there are limited nanomedicine efforts for chronic kidney diseases (CKDs), which affect 844 million people worldwide and can be linked to a variety of genetic kidney diseases. In this Perspective, we provide a brief overview of the clinical status of genetic kidney diseases, background on kidney physiology and a summary of nanoparticle design that enable kidney access and targeting, and emerging technological strategies that can be applied for genetic kidney diseases, including rare and congenital kidney diseases. Finally, we conclude by discussing gaps in knowledge remaining in both genetic kidney diseases and kidney nanomedicine and collective efforts that are needed to bring together stakeholders from diverse expertise and industries to enable the development of the most relevant drug delivery strategies that can make an impact in the clinic.

摘要

自 20 世纪 90 年代以来,纳米颗粒作为药物输送载体已经造福于包括癌症在内的多种疾病,最近,它们在新冠疫情等全球疾病中迅速、高效地被动员起来的潜力已经得到证实。尽管有这些成功的案例,但针对慢性肾病(CKD)的纳米医学研究却很有限,全世界有 8.44 亿人受到慢性肾病的影响,而慢性肾病可能与多种遗传性肾脏疾病有关。在本观点中,我们简要概述了遗传性肾脏疾病的临床现状、肾脏生理学背景以及能够实现肾脏进入和靶向的纳米颗粒设计概述,并总结了可应用于遗传性肾脏疾病(包括罕见和先天性肾脏疾病)的新兴技术策略。最后,我们通过讨论遗传性肾脏疾病和肾脏纳米医学中仍然存在的知识差距,并讨论需要共同努力将来自不同专业知识和行业的利益相关者聚集在一起,以制定最相关的药物输送策略,从而在临床上产生影响,来结束本文。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6094/10145694/f20a4215f1af/nn2c12140_0001.jpg

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