Enzyme replacement therapy with recombinant pro-CTSD (cathepsin D) corrects defective proteolysis and autophagy in neuronal ceroid lipofuscinosis

• Published in: Autophagy Vol. 16; no. 5; pp. 811 - 825
• Main Authors: Marques, André R. A., Di Spiezio, Alessandro, Thießen, Niklas, Schmidt, Lina, Grötzinger, Joachim, Lüllmann-Rauch, Renate, Damme, Markus, Storck, Steffen E., Pietrzik, Claus U., Fogh, Jens, Bär, Julia, Mikhaylova, Marina, Glatzel, Markus, Bassal, Mahmoud, Bartsch, Udo, Saftig, Paul
• Format: Journal Article
• Language: English
• Published: United States Taylor & Francis 03.05.2020
• Subjects: Autophagy; cathepsin D; enzyme replacement therapy; lysosome; neuronal ceroid lipofuscinosis; proteolysis; Research Paper; storage; therapy; enzyme replacement therapy; cathepsin D; therapy; storage; neuronal ceroid lipofuscinosis; Autophagy; lysosome; proteolysis

CTSD (cathepsin D) is one of the major lysosomal proteases indispensable for the maintenance of cellular proteostasis by turning over substrates of endocytosis, phagocytosis and autophagy. Consequently, CTSD deficiency leads to a strong impairment of the lysosomal-autophagy machinery. In mice and hu...