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pro vyhledávání: '"Carmen Garnacho"'
Autor:
Carmen Garnacho, Silvia Muro
Publikováno v:
Journal of Drug Targeting. 25:786-795
Enzyme replacement is a viable treatment for diseases caused by genetic deficiency of lysosomal enzymes. However, suboptimal access of enzymes to target sites limits this strategy. Polymer nanocarriers (NCs) coated with antibody against intercellular
A Comparative Study on the Alterations of Endocytic Pathways in Multiple Lysosomal Storage Disorders
Publikováno v:
Molecular Pharmaceutics. 13:357-368
Many cellular activities and pharmaceutical interventions involve endocytosis and delivery to lysosomes for processing. Hence, lysosomal processing defects can cause cell and tissue damage, as in lysosomal storage diseases (LSDs) characterized by lys
Publikováno v:
Molecular Pharmaceutics
Drugs often use endocytosis to achieve intracellular delivery, either by passive uptake from the extracellular fluid or by active targeting of cell surface features such as endocytic receptors. An example is enzyme replacement therapy, a clinically p
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy. 25(7)
Acid sphingomyelinase deficiency in type B Niemann-Pick disease leads to lysosomal sphingomyelin storage, principally affecting lungs, liver, and spleen. Infused recombinant enzyme is beneficial, yet its delivery to the lungs is limited and requires
Autor:
Yang Shen, Tridib Kumar Bhowmick, Carmen Garnacho, Daniel Serrano, Janet Hsu, Yuan-Chia Kuo, Silvia Muro, Kishan Kumar
Publikováno v:
Journal of Controlled Release. 149:323-331
Fabry disease, due to the deficiency of α-galactosidase A (α-Gal), causes lysosomal accumulation of globotriaosylceramide (Gb3) in multiple tissues and prominently in the vascular endothelium. Although enzyme replacement therapy (ERT) by injection
Autor:
Silvia Muro, Carmen Garnacho, Jason Papademetriou, Edward H. Schuchman, Daniel Serrano, Tridib Kumar Bhowmick
Publikováno v:
Journal of inherited metabolic disease. 36(3)
Targeting lysosomal enzymes to receptors involved in transport into and across cells holds promise to enhance peripheral and brain delivery of enzyme replacement therapies (ERTs) for lysosomal storage disorders. Receptors being explored include those