The DOI matches its Crossref record; 309 of the 309 references it cites were found in a registry or an index.
Lysosomes as a therapeutic target
Open accessAbstract
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. Defective lysosomal function has been implicated in diseases ranging from rare lysosomal storage disorders to more common diseases including inflammatory and autoimmune disorders, neurodegenerative diseases, cancer and metabolic disorders. Here, Muller and colleagues provide an overview of the physiological and pathological roles of lysosomes and assess the progress and opportunities for therapeutically targeting lysosomal proteins and processes.
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Cited by
Mechanisms of autophagy–lysosome dysfunction in neurodegenerative diseases
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Metadata
- Authors
- Bonam, S. R., Wang, F., & Muller, S.
- Venue
- Nature Reviews Drug Discovery
- Published
- 2019-09-02
- Volume
- 18
- Issue
- 12
- Pages
- 923-948
- ISSN
- 1474-1776 (ISSN-L)
- 1474-1784
- OpenAlex
- W2971120492
- Cited by
- 759
- References
- 309
- FWCI
- 14.30
- Type
- journal article
- Language
- en
- Open access
- bronze
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