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- Title
Nanoformulation of Therapeutic Enzymes: A Short Review.
- Authors
Tóth, Gergő Dániel; Koplányi, Gábor; Kenéz, Balázs; Balogh-Weiser, Diána
- Abstract
Enzyme replacement therapy (ERT) is a therapeutic approach that involves the administration of specific enzymes to the patient in order to correct metabolic defects caused by enzyme deficiency. The formulation of ERTs involves the production, purification, and formulation of the enzyme into a stable and biologically active drug product, often using recombinant DNA technology. Non-systemic ERTs often involve the immobilization of the enzyme on a carrier, such as hydrogels, liposomes, or nanoparticles. ERT holds great promise for the treatment of a wide range of genetic disorders, and its success regarding lysosomal storage diseases, such as Fabry disease, Gaucher disease, and Pompe disease has paved the way for the development of similar therapies for other genetic disorders too.
- Subjects
LYSOSOMAL storage diseases; ENZYME replacement therapy; GLYCOGEN storage disease type II; ENZYME deficiency; ANGIOKERATOMA corporis diffusum
- Publication
Periodica Polytechnica: Chemical Engineering, 2023, Vol 67, Issue 4, p624
- ISSN
0324-5853
- Publication type
Article
- DOI
10.3311/PPch.22826