Antibody-Mediated Enzyme Therapeutics and Applications in Glycogen Storage Diseases

Zhengqiu Zhou, Grant L. Austin, Robert Shaffer, Dustin D. Armstrong, Matthew S. Gentry

Research output: Contribution to journalReview articlepeer-review

22 Scopus citations


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 languageEnglish
Pages (from-to)1094-1109
Number of pages16
JournalTrends in Molecular Medicine
Issue number12
StatePublished - Dec 2019

Bibliographical note

Publisher Copyright:
© 2019 Elsevier Ltd


  • Pompe disease
  • antibody-directed therapies, Lafora disease
  • antibody–drug conjugate
  • antibody–enzyme fusion
  • glycogen

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology


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