Antibody-Mediated Enzyme Therapeutics and Applications in Glycogen Storage Diseases

Trends Mol Med. 2019 Dec;25(12):1094-1109. doi: 10.1016/j.molmed.2019.08.005. Epub 2019 Sep 12.


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.

Keywords: Pompe disease; antibody-directed therapies, Lafora disease; antibody–drug conjugate; antibody–enzyme fusion; glycogen.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Antibodies / administration & dosage
  • Antibodies / therapeutic use*
  • Enzyme Therapy / methods*
  • Glycogen Storage Disease / drug therapy*
  • Humans
  • Immunoconjugates / administration & dosage
  • Immunoconjugates / therapeutic use
  • Recombinant Fusion Proteins / administration & dosage
  • Recombinant Fusion Proteins / therapeutic use


  • Antibodies
  • Immunoconjugates
  • Recombinant Fusion Proteins