Targeting lysosomes in human disease: from basic research to clinical applications

Abstract In recent years, accumulating evidence has elucidated the role of lysosomes in dynamically regulating cellular and organismal homeostasis. Lysosomal changes and dysfunction have been correlated with the development of numerous diseases. In this review, we interpreted the key biological func...

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Autores principales: Mengdie Cao, Xiangyuan Luo, Kongming Wu, Xingxing He
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Lenguaje:EN
Publicado: Nature Publishing Group 2021
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Acceso en línea:https://doaj.org/article/f9ff10764de34d2ea8167d4ef4929b0c
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spelling oai:doaj.org-article:f9ff10764de34d2ea8167d4ef4929b0c2021-11-14T12:10:50ZTargeting lysosomes in human disease: from basic research to clinical applications10.1038/s41392-021-00778-y2059-3635https://doaj.org/article/f9ff10764de34d2ea8167d4ef4929b0c2021-11-01T00:00:00Zhttps://doi.org/10.1038/s41392-021-00778-yhttps://doaj.org/toc/2059-3635Abstract In recent years, accumulating evidence has elucidated the role of lysosomes in dynamically regulating cellular and organismal homeostasis. Lysosomal changes and dysfunction have been correlated with the development of numerous diseases. In this review, we interpreted the key biological functions of lysosomes in four areas: cellular metabolism, cell proliferation and differentiation, immunity, and cell death. More importantly, we actively sought to determine the characteristic changes and dysfunction of lysosomes in cells affected by these diseases, the causes of these changes and dysfunction, and their significance to the development and treatment of human disease. Furthermore, we outlined currently available targeting strategies: (1) targeting lysosomal acidification; (2) targeting lysosomal cathepsins; (3) targeting lysosomal membrane permeability and integrity; (4) targeting lysosomal calcium signaling; (5) targeting mTOR signaling; and (6) emerging potential targeting strategies. Moreover, we systematically summarized the corresponding drugs and their application in clinical trials. By integrating basic research with clinical findings, we discussed the current opportunities and challenges of targeting lysosomes in human disease.Mengdie CaoXiangyuan LuoKongming WuXingxing HeNature Publishing GrouparticleMedicineRBiology (General)QH301-705.5ENSignal Transduction and Targeted Therapy, Vol 6, Iss 1, Pp 1-28 (2021)
institution DOAJ
collection DOAJ
language EN
topic Medicine
R
Biology (General)
QH301-705.5
spellingShingle Medicine
R
Biology (General)
QH301-705.5
Mengdie Cao
Xiangyuan Luo
Kongming Wu
Xingxing He
Targeting lysosomes in human disease: from basic research to clinical applications
description Abstract In recent years, accumulating evidence has elucidated the role of lysosomes in dynamically regulating cellular and organismal homeostasis. Lysosomal changes and dysfunction have been correlated with the development of numerous diseases. In this review, we interpreted the key biological functions of lysosomes in four areas: cellular metabolism, cell proliferation and differentiation, immunity, and cell death. More importantly, we actively sought to determine the characteristic changes and dysfunction of lysosomes in cells affected by these diseases, the causes of these changes and dysfunction, and their significance to the development and treatment of human disease. Furthermore, we outlined currently available targeting strategies: (1) targeting lysosomal acidification; (2) targeting lysosomal cathepsins; (3) targeting lysosomal membrane permeability and integrity; (4) targeting lysosomal calcium signaling; (5) targeting mTOR signaling; and (6) emerging potential targeting strategies. Moreover, we systematically summarized the corresponding drugs and their application in clinical trials. By integrating basic research with clinical findings, we discussed the current opportunities and challenges of targeting lysosomes in human disease.
format article
author Mengdie Cao
Xiangyuan Luo
Kongming Wu
Xingxing He
author_facet Mengdie Cao
Xiangyuan Luo
Kongming Wu
Xingxing He
author_sort Mengdie Cao
title Targeting lysosomes in human disease: from basic research to clinical applications
title_short Targeting lysosomes in human disease: from basic research to clinical applications
title_full Targeting lysosomes in human disease: from basic research to clinical applications
title_fullStr Targeting lysosomes in human disease: from basic research to clinical applications
title_full_unstemmed Targeting lysosomes in human disease: from basic research to clinical applications
title_sort targeting lysosomes in human disease: from basic research to clinical applications
publisher Nature Publishing Group
publishDate 2021
url https://doaj.org/article/f9ff10764de34d2ea8167d4ef4929b0c
work_keys_str_mv AT mengdiecao targetinglysosomesinhumandiseasefrombasicresearchtoclinicalapplications
AT xiangyuanluo targetinglysosomesinhumandiseasefrombasicresearchtoclinicalapplications
AT kongmingwu targetinglysosomesinhumandiseasefrombasicresearchtoclinicalapplications
AT xingxinghe targetinglysosomesinhumandiseasefrombasicresearchtoclinicalapplications
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