TY - JOUR
T1 - Lysosomes as a therapeutic target
AU - Bonam, Srinivasa Reddy
AU - Wang, Fengjuan
AU - Muller, Sylviane
N1 - Funding Information:
The authors apologize to all those whose work is not cited due to space limitations. They gratefully acknowledge Hélène Jeltsch-David for critically reading the manuscript. This research was funded by the French Centre National de la Recherche Scientifique, Région Alsace, the Laboratory of Excellence Medalis (ANR-10-LABX-0034), Initiative of Excellence (IdEx), Strasbourg University, and ImmuPharma France. S.M. is grateful to the University of Strasbourg Institute for Advanced Study (USIAS) for funding F.W., and acknowledges the support of the TRANSAUTOPHAGY COST Action (CA15138), the Club francophone de l’autophagie (CFATG) and the European Regional Development Fund of the European Union in the framework of the INTERREG V Upper Rhine programme.
Publisher Copyright:
© 2019, Springer Nature Limited.
PY - 2019/12/1
Y1 - 2019/12/1
N2 - Lysosomes are membrane-bound organelles with roles in processes involved in degrading and recycling cellular waste, cellular signalling and energy metabolism. Defects in genes encoding lysosomal proteins cause lysosomal storage disorders, in which enzyme replacement therapy has proved successful. Growing evidence also implicates roles for lysosomal dysfunction in more common diseases including inflammatory and autoimmune disorders, neurodegenerative diseases, cancer and metabolic disorders. With a focus on lysosomal dysfunction in autoimmune disorders and neurodegenerative diseases — including lupus, rheumatoid arthritis, multiple sclerosis, Alzheimer disease and Parkinson disease — this Review critically analyses progress and opportunities for therapeutically targeting lysosomal proteins and processes, particularly with small molecules and peptide drugs.
AB - Lysosomes are membrane-bound organelles with roles in processes involved in degrading and recycling cellular waste, cellular signalling and energy metabolism. Defects in genes encoding lysosomal proteins cause lysosomal storage disorders, in which enzyme replacement therapy has proved successful. Growing evidence also implicates roles for lysosomal dysfunction in more common diseases including inflammatory and autoimmune disorders, neurodegenerative diseases, cancer and metabolic disorders. With a focus on lysosomal dysfunction in autoimmune disorders and neurodegenerative diseases — including lupus, rheumatoid arthritis, multiple sclerosis, Alzheimer disease and Parkinson disease — this Review critically analyses progress and opportunities for therapeutically targeting lysosomal proteins and processes, particularly with small molecules and peptide drugs.
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U2 - 10.1038/s41573-019-0036-1
DO - 10.1038/s41573-019-0036-1
M3 - Review article
C2 - 31477883
AN - SCOPUS:85072108585
SN - 1474-1776
VL - 18
SP - 923
EP - 948
JO - Nature Reviews Drug Discovery
JF - Nature Reviews Drug Discovery
IS - 12
ER -