Antibody-Mediated Enzyme Therapeutics and Applications in Glycogen Storage Diseases

Overview

Journal Trends Mol Med

Specialties General Medicine
Molecular Biology

Date 2019 Sep 17

PMID 31522955

Citations 20

Authors

Zhengqiu Zhou

Grant L Austin

Robert Shaffer

Dustin D Armstrong

Matthew S Gentry

Affiliations

Soon will be listed here.

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.

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