Results 1 to 10 of about 686,065 (200)

Engineering of GlcNAc-1-Phosphotransferase for Production of Highly Phosphorylated Lysosomal Enzymes for Enzyme Replacement Therapy [PDF]

Molecular Therapy: Methods & Clinical Development, 2017
Several lysosomal enzymes currently used for enzyme replacement therapy in patients with lysosomal storage diseases contain very low levels of mannose 6-phosphate, limiting their uptake via mannose 6-phosphate receptors on the surface of the deficient ...
Lin Liu, Wang-Sik Lee, Balraj Doray, Stuart Kornfeld   +3 more
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A universal GlycoDesign for lysosomal replacement enzymes to improve circulation time and biodistribution [PDF]

Frontiers in Bioengineering and Biotechnology, 2023
Currently available enzyme replacement therapies for lysosomal storage diseases are limited in their effectiveness due in part to short circulation times and suboptimal biodistribution of the therapeutic enzymes.
Yen-Hsi Chen, Yen-Hsi Chen, Weihua Tian, Weihua Tian, Makiko Yasuda, Zilu Ye, Zilu Ye, Ming Song, Ulla Mandel, Claus Kristensen, Lorenzo Povolo, André R. A. Marques, Tomislav Čaval, Tomislav Čaval, Albert J. R. Heck, Julio Lopes Sampaio, Ludger Johannes, Takahiro Tsukimura, Takahiro Tsukimura, Robert Desnick, Sergey Y. Vakhrushev, Zhang Yang, Zhang Yang, Henrik Clausen   +23 more
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GRASP55 maintains lysosome function by controlling sorting of lysosomal enzymes at the Golgi [PDF]

EMBO Reports
Lysosomes are multifunctional organelles that play important roles in cellular recycling, signaling, and homeostasis, relying on precise trafficking and activation of lysosomal enzymes.
Julian Nüchel, Maryam Omidi, Stephanie A Fernandes, Marina Tauber, Sandra Pohl, Markus Plomann, Constantinos Demetriades   +6 more
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Lysosomal enzymes and mannose 6-phosphate receptors in the lacrimal drainage system: Evidence and its potential implications [PDF]

Indian Journal of Ophthalmology, 2018
Purpose: To investigate the presence and patterns of lysosomal enzymes and mannose 6-phosophate receptor (MPRs) in human lacrimal drainage system. Methods: The study was performed on healthy lacrimal sacs and nasolacrimal ducts obtained from exenteration
Mohammad Javed Ali, Ashapogu Venugopal, Kavyashree Sakharayapatna Ranganath, Nadimpalli Siva Kumar   +3 more
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Progranulin acts as a shared chaperone and regulates multiple lysosomal enzymes [PDF]

Genes and Diseases, 2017
Multifunctional factor progranulin (PGRN) plays an important role in lysosomes, and its mutations and insufficiency are associated with lysosomal storage diseases, including neuronal ceroid lipofuscinosis and Gaucher disease (GD).
Jinlong Jian, Aubryanna Hettinghouse, Chuan-ju Liu   +2 more
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Extracellular vesicles from recombinant cell factories improve the activity and efficacy of enzymes defective in lysosomal storage disorders

Journal of Extracellular Vesicles, 2021
In the present study the use of extracellular vesicles (EVs) as vehicles for therapeutic enzymes in lysosomal storage disorders was explored. EVs were isolated from mammalian cells overexpressing alpha‐galactosidase A (GLA) or N‐sulfoglucosamine ...
Joaquin Seras‐Franzoso, Zamira V. Díaz‐Riascos, José Luis Corchero, Patricia González, Natalia García‐Aranda, Mònica Mandaña, Roger Riera, Ana Boullosa, Sandra Mancilla, Alba Grayston, Marc Moltó‐Abad, Elena Garcia‐Fruitós, Rosa Mendoza, Guillem Pintos‐Morell, Lorenzo Albertazzi, Anna Rosell, Josefina Casas, Antonio Villaverde, Simó Schwartz Jr, Ibane Abasolo   +19 more
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Biomarkers of oxidative stress, biochemical changes, and the activity of lysosomal enzymes in the livers of rainbow trout (Oncorhynchus mykiss Walbaum) vaccinated against yersiniosis before a Yersinia ruckeri challenge [PDF]

Journal of Veterinary Research
This study aimed to evaluate biomarkers of oxidative stress (2-thiobarbituric acid reactive substances, aldehyde and ketone derivatives of oxidatively modified proteins and total antioxidant capacity), the activity of antioxidant enzymes (superoxide ...
Kurhaluk Natalia, Grudniewska Joanna, Pękala-Safińska Agnieszka, Pajdak-Czaus Joanna, Terech-Majewska Elżbieta, Platt-Samoraj Aleksandra, Tkaczenko Halina   +6 more
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Lysosomal storage diseases [PDF]

Journal of Veterinary and Animal Sciences, 2021
Lysosomes play a pivotal role in cellular processes through an active interplay of enzymes, lysosomal membrane proteins, and cytosolic proteins. Lysosomal storage diseases are a group of inherited and acquired disorders.
Smitha Rose Georgy
doaj   +1 more source

Loss of the batten disease protein CLN3 leads to mis-trafficking of M6PR and defective autophagic-lysosomal reformation

Nature Communications, 2023
Batten disease, one of the most devastating types of neurodegenerative lysosomal storage disorders, is caused by mutations in CLN3. Here, we show that CLN3 is a vesicular trafficking hub connecting the Golgi and lysosome compartments.
Alessia Calcagni’, Leopoldo Staiano, Nicolina Zampelli, Nadia Minopoli, Niculin J. Herz, Giuseppe Di Tullio, Tuong Huynh, Jlenia Monfregola, Alessandra Esposito, Carmine Cirillo, Aleksandar Bajic, Mahla Zahabiyon, Rachel Curnock, Elena Polishchuk, Luke Parkitny, Diego Luis Medina, Nunzia Pastore, Peter J. Cullen, Giancarlo Parenti, Maria Antonietta De Matteis, Paolo Grumati, Andrea Ballabio   +21 more
doaj   +1 more source

Mannose-6-phosphate glycan for lysosomal targeting: various applications from enzyme replacement therapy to lysosome-targeting chimeras

Animal Cells and Systems, 2022
A lysosome, an acidic membrane-bound organelle, contains hydrolytic enzymes to digest macromolecules for recycling. Many lysosomal enzymes (LEs) traffic to the lysosome through the mannose-6-phosphate (M6P)-dependent pathway.
Jinho Seo, Doo-Byoung Oh
doaj   +1 more source