Abstract
The use of antibodies as targeting molecules or cell-penetrating tools has emerged at the forefront of pharmaceutical research. Antibody-directed therapies in the form of antibody–drug conjugates, immune modulators, and antibody-directed enzyme prodrugs have been most extensively utilized as hematological, rheumatological, and oncological therapies, but recent developments are identifying additional applications of antibody-mediated delivery systems. A novel application of this technology is for the treatment of glycogen storage disorders (GSDs) via an antibody–enzyme fusion (AEF) platform to penetrate cells and deliver an enzyme to the cytoplasm, nucleus, and/or other organelles. Exciting developments are currently underway for AEFs in the treatment of the GSDs Pompe disease and Lafora disease (LD). Antibody-based therapies are quickly becoming an integral part of modern disease therapeutics.
Original language | English |
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Pages (from-to) | 1094-1109 |
Number of pages | 16 |
Journal | Trends in Molecular Medicine |
Volume | 25 |
Issue number | 12 |
DOIs | |
State | Published - Dec 2019 |
Bibliographical note
Publisher Copyright:© 2019 Elsevier Ltd
Keywords
- Pompe disease
- antibody-directed therapies, Lafora disease
- antibody–drug conjugate
- antibody–enzyme fusion
- glycogen
ASJC Scopus subject areas
- Molecular Medicine
- Molecular Biology