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

2 Citations (Scopus)

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

Fingerprint

Lafora Disease
Antibodies
Immunoglobulin Isotypes
Enzymes
Glycogen
Epilepsy
Brain
Pharmaceutical Preparations

Keywords

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

ASJC Scopus subject areas

  • Physiology
  • Molecular Biology
  • Cell Biology

Cite this

Targeting Pathogenic Lafora Bodies in Lafora Disease Using an Antibody-Enzyme Fusion. / Brewer, M. Kathryn; Uittenbogaard, Annette; Austin, Grant L.; Segvich, Dyann M.; DePaoli-Roach, Anna; Roach, Peter J.; McCarthy, John J.; Simmons, Zoe R.; Brandon, Jason A.; Zhou, Zhengqiu; Zeller, Jill; Young, Lyndsay E.A.; Sun, Ramon C.; Pauly, James R.; Aziz, Nadine M.; Hodges, Bradley L.; McKnight, Tracy R.; Armstrong, Dustin D.; Gentry, Matthew S.

In: Cell Metabolism, Vol. 30, No. 4, 01.10.2019, p. 689-705.e6.

Research output: Contribution to journalArticle

Brewer, MK, Uittenbogaard, A, Austin, GL, Segvich, DM, DePaoli-Roach, A, Roach, PJ, McCarthy, JJ, Simmons, ZR, Brandon, JA, Zhou, Z, Zeller, J, Young, LEA, Sun, RC, Pauly, JR, Aziz, NM, Hodges, BL, McKnight, TR, Armstrong, DD & Gentry, MS 2019, 'Targeting Pathogenic Lafora Bodies in Lafora Disease Using an Antibody-Enzyme Fusion', Cell Metabolism, vol. 30, no. 4, pp. 689-705.e6. https://doi.org/10.1016/j.cmet.2019.07.002
Brewer, M. Kathryn ; Uittenbogaard, Annette ; Austin, Grant L. ; Segvich, Dyann M. ; DePaoli-Roach, Anna ; Roach, Peter J. ; McCarthy, John J. ; Simmons, Zoe R. ; Brandon, Jason A. ; Zhou, Zhengqiu ; Zeller, Jill ; Young, Lyndsay E.A. ; Sun, Ramon C. ; Pauly, James R. ; Aziz, Nadine M. ; Hodges, Bradley L. ; McKnight, Tracy R. ; Armstrong, Dustin D. ; Gentry, Matthew S. / Targeting Pathogenic Lafora Bodies in Lafora Disease Using an Antibody-Enzyme Fusion. In: Cell Metabolism. 2019 ; Vol. 30, No. 4. pp. 689-705.e6.
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