Generation of two human iPSC lines, FINCBi002-A and FINCBi003-A, carrying heteroplasmic macrodeletion of mitochondrial DNA causing Pearson's syndrome.


Journal

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

Informations de publication

Date de publication:
04 Jan 2021
Historique:
received: 20 10 2020
revised: 03 12 2020
accepted: 30 12 2020
pubmed: 13 1 2021
medline: 13 1 2021
entrez: 12 1 2021
Statut: aheadofprint

Résumé

Pearson marrow pancreas syndrome (PMPS) is a sporadic mitochondrial disease, resulting from the clonal expansion of a mutated mitochondrial DNA (mtDNA) molecule bearing a macro-deletion, and therefore missing essential genetic information. PMPS is characterized by the presence of deleted (Δ) mtDNA that co-exist with the presence of a variable amount of wild-type mtDNA, a condition termed heteroplasmy. All tissues of the affected individual, including the haemopoietic system and the post-mitotic, highly specialized tissues (brain, skeletal muscle, and heart) contain the large-scale mtDNA deletion in variable amount. We generated human induced pluripotent stem cells (hiPSCs) from two PMPS patients, carrying different type of large-scale deletion.

Identifiants

pubmed: 33434818
pii: S1873-5061(20)30452-9
doi: 10.1016/j.scr.2020.102151
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

102151

Informations de copyright

Copyright © 2020 The Author(s). Published by Elsevier B.V. All rights reserved.

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.

Angelo Iannielli (A)

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

Andrea Cavaliere (A)

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

Andrea Legati (A)

Unit of Medical Genetics and Neurogenetics, Fondazione IRCCS Istituto Neurologico "Carlo Besta", 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)

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

Classifications MeSH