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

doi:10.1016/j.cmet.2019.07.002

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

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63 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 language	English
Pages (from-to)	689-705.e6
Journal	Cell Metabolism
Volume	30
Issue number	4
DOIs	https://doi.org/10.1016/j.cmet.2019.07.002
State	Published - 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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10.1016/j.cmet.2019.07.002

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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

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title = "Targeting Pathogenic Lafora Bodies in Lafora Disease Using an Antibody-Enzyme Fusion",

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.",

keywords = "Lafora bodies, Lafora disease, amylase, antibody-based drug, antibody-enzyme fusion, enzyme therapy, epilepsy, glycogen, glycogen storage disease, metabolomics",

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

note = "Publisher Copyright: {\textcopyright} 2019 Elsevier Inc.",

year = "2019",

month = oct,

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doi = "10.1016/j.cmet.2019.07.002",

language = "English",

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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

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AU - Brewer, M. Kathryn

AU - Uittenbogaard, Annette

AU - Austin, Grant L.

AU - Segvich, Dyann M.

AU - DePaoli-Roach, Anna

AU - Roach, Peter J.

AU - McCarthy, John J.

AU - Simmons, Zoe R.

AU - Brandon, Jason A.

AU - Zhou, Zhengqiu

AU - Zeller, Jill

AU - Young, Lyndsay E.A.

AU - Sun, Ramon C.

AU - Pauly, James R.

AU - Aziz, Nadine M.

AU - Hodges, Bradley L.

AU - McKnight, Tracy R.

AU - Armstrong, Dustin D.

AU - Gentry, Matthew S.

PY - 2019/10/1

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AB - 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.

KW - Lafora bodies

KW - Lafora disease

KW - amylase

KW - antibody-based drug

KW - antibody-enzyme fusion

KW - enzyme therapy

KW - epilepsy

KW - glycogen

KW - glycogen storage disease

KW - metabolomics

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JO - Cell Metabolism

JF - Cell Metabolism

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

Abstract

Bibliographical note

Keywords

ASJC Scopus subject areas

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