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 journalArticlepeer-review

59 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
Pages (from-to)689-705.e6
JournalCell Metabolism
Volume30
Issue number4
DOIs
StatePublished - Oct 1 2019

Bibliographical note

Publisher Copyright:
© 2019 Elsevier Inc.

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

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