Targeting Pathogenic Lafora Bodies in Lafora Disease Using an Antibody-Enzyme Fusion

M. Kathryn Brewer, Annette Uittenbogaard, Grant L. Austin, Dyann M. Segvich, Anna DePaoli-Roach, Peter J. Roach, John J. McCarthy, Zoe R. Simmons, Jason A. Brandon, Zhengqiu Zhou, Jill Zeller, Lyndsay E.A. Young, Ramon C. Sun, James R. Pauly, Nadine M. Aziz, Bradley L. Hodges, Tracy R. McKnight, Dustin D. Armstrong, Matthew S. Gentry

Research output: Contribution to journalArticle

4 Scopus citations

Abstract

Lafora disease (LD) is a devastating childhood epilepsy caused by intracellular, aberrant glycogen aggregates called Lafora bodies (LBs) in the brain and other tissues. Herein, Brewer et al. generated a first-in-class antibody-enzyme fusion, VAL-0417, that degrades LBs in vitro and in vivo in a pre-clinical model, showing promise as a LD drug.

Original languageEnglish (US)
Pages (from-to)689-705.e6
JournalCell Metabolism
Volume30
Issue number4
DOIs
StatePublished - Oct 1 2019

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Keywords

  • Lafora bodies
  • Lafora disease
  • amylase
  • antibody-based drug
  • antibody-enzyme fusion
  • enzyme therapy
  • epilepsy
  • glycogen
  • glycogen storage disease
  • metabolomics

ASJC Scopus subject areas

  • Physiology
  • Molecular Biology
  • Cell Biology

Cite this

Brewer, M. K., Uittenbogaard, A., Austin, G. L., Segvich, D. M., DePaoli-Roach, A., Roach, P. J., McCarthy, J. J., Simmons, Z. R., Brandon, J. A., Zhou, Z., Zeller, J., Young, L. E. A., Sun, R. C., Pauly, J. R., Aziz, N. M., Hodges, B. L., McKnight, T. R., Armstrong, D. D., & Gentry, M. S. (2019). Targeting Pathogenic Lafora Bodies in Lafora Disease Using an Antibody-Enzyme Fusion. Cell Metabolism, 30(4), 689-705.e6. https://doi.org/10.1016/j.cmet.2019.07.002