Generation of a human iPSC line, FINCBi001-A, carrying a homoplasmic m.G3460A mutation in MT-ND1 associated with Leber's Hereditary optic Neuropathy (LHON).


Journal

Stem cell research
ISSN: 1876-7753
Titre abrégé: Stem Cell Res
Pays: England
ID NLM: 101316957

Informations de publication

Date de publication:
10 2020
Historique:
received: 08 07 2020
revised: 23 07 2020
accepted: 28 07 2020
pubmed: 11 8 2020
medline: 22 6 2021
entrez: 11 8 2020
Statut: ppublish

Résumé

Leber's Hereditary Optic Neuropathy (LHON) is a maternally inherited disorder caused by homoplasmic mutations of mitochondrial DNA (mtDNA). LHON is characterized by the selective degeneration of the retinal ganglion cells (RGC). Almost all LHON maternal lineages are homoplasmic mutant (100% mtDNA copies are mutant) for one of three frequent mtDNA mutations now found in over 90% of patients worldwide (m.11778G > A/MT-ND4, m.3460G > A/MT-ND1, m.14484 T > C/MT-ND6). Human induced pluripotent stem cells (hiPSCs) were generated from a patient carrying the homoplasmic m.3460G > A/MT-ND1 mutation using the Sendai virus non-integrating virus.

Identifiants

pubmed: 32771908
pii: S1873-5061(20)30240-3
doi: 10.1016/j.scr.2020.101939
pii:
doi:

Substances chimiques

DNA, Mitochondrial 0
NADH Dehydrogenase EC 1.6.99.3
MT-ND1 protein, human EC 7.1.1.2

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

101939

Informations de copyright

Copyright © 2020 The Authors. Published by Elsevier B.V. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Auteurs

Camille Peron (C)

Unit of Medical Genetics and Neurogenetics, Fondazione IRCCS Istituto Neurologico "Carlo Besta", Milan, Italy.

Roberta Mauceri (R)

Unit of Medical Genetics and Neurogenetics, Fondazione IRCCS Istituto Neurologico "Carlo Besta", Milan, Italy.

Tommaso Cabassi (T)

Unit of Medical Genetics and Neurogenetics, Fondazione IRCCS Istituto Neurologico "Carlo Besta", Milan, Italy.

Alice Segnali (A)

Unit of Medical Genetics and Neurogenetics, Fondazione IRCCS Istituto Neurologico "Carlo Besta", Milan, Italy.

Alessandra Maresca (A)

IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy.

Angelo Iannielli (A)

Division of Neuroscience, San Raffaele Scientific Institute, Milan, Italy; National Research Council (CNR), Institute of Neuroscience, Milan, Italy.

Ambra Rizzo (A)

Laboratory of Clinical Investigation, Fondazione IRCCS Istituto Neurologico "Carlo Besta", Milan, Italy.

Francesca L Sciacca (FL)

Laboratory of Clinical Investigation, Fondazione IRCCS Istituto Neurologico "Carlo Besta", Milan, Italy.

Vania Broccoli (V)

Division of Neuroscience, San Raffaele Scientific Institute, Milan, Italy; National Research Council (CNR), Institute of Neuroscience, Milan, Italy.

Valerio Carelli (V)

IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy; Department of Biomedical and Neuromotor Sciences - DIBINEM, University of Bologna, Bologna, Italy.

Valeria Tiranti (V)

Unit of Medical Genetics and Neurogenetics, Fondazione IRCCS Istituto Neurologico "Carlo Besta", Milan, Italy. Electronic address: valeria.tiranti@istituto-besta.it.

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Classifications MeSH