Fiche publication


Date publication

septembre 2019

Journal

Nature reviews. Drug discovery

Auteurs

Membres identifiés du Cancéropôle Est :
Pr MULLER Sylviane


Tous les auteurs :
Bonam SR, Wang F, Muller S

Résumé

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.

Référence

Nat Rev Drug Discov. 2019 Sep 2;: