An empirical pipeline for personalized diagnosis of Lafora disease mutations.

Biochemistry Biophysics Disease Structural biology

Journal

iScience
ISSN: 2589-0042
Titre abrégé: iScience
Pays: United States
ID NLM: 101724038

Informations de publication

Date de publication:
19 Nov 2021
Historique:
received: 10 08 2021
revised: 14 09 2021
accepted: 12 10 2021
entrez: 10 11 2021
pubmed: 11 11 2021
medline: 11 11 2021
Statut: epublish

Résumé

Lafora disease (LD) is a fatal childhood dementia characterized by progressive myoclonic epilepsy manifesting in the teenage years, rapid neurological decline, and death typically within ten years of onset. Mutations in either

Identifiants

pubmed: 34755096
doi: 10.1016/j.isci.2021.103276
pii: S2589-0042(21)01245-1
pmc: PMC8564118
doi:

Types de publication

Journal Article

Langues

eng

Pagination

103276

Subventions

Organisme : NINDS NIH HHS
ID : R35 NS116824
Pays : United States
Organisme : NCATS NIH HHS
ID : TL1 TR001997
Pays : United States

Informations de copyright

© 2021 The Authors.

Déclaration de conflit d'intérêts

M.S.G. is a consultant for Maze Therapeutics, Enable Therapeutics, Glut1 Deficiency Syndrome Foundation, and Chelsea’s Hope. M.S.G. and C.V.K. are founders of Atterogen, LLC.

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Auteurs

M Kathryn Brewer (MK)

Department of Molecular and Cellular Biochemistry, University of Kentucky College of Medicine, Lexington, KY 40536, USA.
Lafora Epilepsy Cure Initiative, Epilepsy and Brain Metabolism Center, and Center for Structural Biology, University of Kentucky College of Medicine, Lexington, KY 40536, USA.

Maria Machio-Castello (M)

Neurology Laboratory and Epilepsy Unit, Department of Neurology, IIS Fundación Jiménez Díaz, UAM and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid 28040, Spain.

Rosa Viana (R)

Institute of Biomedicine of Valencia, CSIC and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Valencia 46010, Spain.

Jeremiah L Wayne (JL)

Department of Molecular and Cellular Biochemistry, University of Kentucky College of Medicine, Lexington, KY 40536, USA.

Andrea Kuchtová (A)

Department of Molecular and Cellular Biochemistry, University of Kentucky College of Medicine, Lexington, KY 40536, USA.

Zoe R Simmons (ZR)

Department of Molecular and Cellular Biochemistry, University of Kentucky College of Medicine, Lexington, KY 40536, USA.

Sarah Sternbach (S)

Department of Molecular and Cellular Biochemistry, University of Kentucky College of Medicine, Lexington, KY 40536, USA.

Sheng Li (S)

Department of Medicine, University of California at San Diego, La Jolla, CA 92093, USA.

Maria Adelaida García-Gimeno (MA)

Department of Biotechnology, Escuela Técnica Superior de Ingeniería Agronómica y del Medio Natural (ETSIAMN), Polytechnic University of Valencia, 46022 Valencia, Spain.

Jose M Serratosa (JM)

Lafora Epilepsy Cure Initiative, Epilepsy and Brain Metabolism Center, and Center for Structural Biology, University of Kentucky College of Medicine, Lexington, KY 40536, USA.
Neurology Laboratory and Epilepsy Unit, Department of Neurology, IIS Fundación Jiménez Díaz, UAM and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid 28040, Spain.

Pascual Sanz (P)

Lafora Epilepsy Cure Initiative, Epilepsy and Brain Metabolism Center, and Center for Structural Biology, University of Kentucky College of Medicine, Lexington, KY 40536, USA.
Institute of Biomedicine of Valencia, CSIC and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Valencia 46010, Spain.

Craig W Vander Kooi (CW)

Department of Molecular and Cellular Biochemistry, University of Kentucky College of Medicine, Lexington, KY 40536, USA.
Lafora Epilepsy Cure Initiative, Epilepsy and Brain Metabolism Center, and Center for Structural Biology, University of Kentucky College of Medicine, Lexington, KY 40536, USA.

Matthew S Gentry (MS)

Department of Molecular and Cellular Biochemistry, University of Kentucky College of Medicine, Lexington, KY 40536, USA.
Lafora Epilepsy Cure Initiative, Epilepsy and Brain Metabolism Center, and Center for Structural Biology, University of Kentucky College of Medicine, Lexington, KY 40536, USA.

Classifications MeSH