| Literature DB >> 31522955 |
Zhengqiu Zhou1, Grant L Austin1, Robert Shaffer2, Dustin D Armstrong2, Matthew S Gentry3.
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.Entities:
Keywords: Pompe disease; antibody-directed therapies, Lafora disease; antibody–drug conjugate; antibody–enzyme fusion; glycogen
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Year: 2019 PMID： 31522955 PMCID： PMC6889062 DOI： 10.1016/j.molmed.2019.08.005
Source DB: PubMed Journal: Trends Mol Med ISSN： 1471-4914 Impact factor: 11.951