In Vitro Disease Modeling of Hermansky-Pudlak Syndrome Type 2 Using Human Induced Pluripotent Stem Cell-Derived Alveolar Organoids.
HPS
Hermansky-Pudlak syndrome
MX35
alveolar organoid
alveolar type 2 cell
iPSC
lamellar body
pluripotent stem cell
pulmonary fibrosis
pulmonary surfactant
Journal
Stem cell reports
ISSN: 2213-6711
Titre abrégé: Stem Cell Reports
Pays: United States
ID NLM: 101611300
Informations de publication
Date de publication:
05 03 2019
05 03 2019
Historique:
received:
01
06
2018
revised:
17
01
2019
accepted:
17
01
2019
pubmed:
19
2
2019
medline:
7
3
2020
entrez:
19
2
2019
Statut:
ppublish
Résumé
It has been challenging to generate in vitro models of alveolar lung diseases, as the stable culture of alveolar type 2 (AT2) cells has been difficult. Methods of generating and expanding AT2 cells derived from induced pluripotent stem cells (iPSCs) have been established and are expected to be applicable to disease modeling. Hermansky-Pudlak syndrome (HPS) is an autosomal recessive disorder characterized by dysfunction of lysosome-related organelles, such as lamellar bodies (LBs), in AT2 cells. From an HPS type 2 (HPS2) patient, we established disease-specific iPSCs (HPS2-iPSCs) and their gene-corrected counterparts. By live cell imaging, the LB dynamics were visualized and altered distribution, enlargement, and impaired secretion of LBs were demonstrated in HPS2-iPSC-derived AT2 cells. These findings provide insight into the AT2 dysfunction in HPS patients and support the potential use of human iPSC-derived AT2 cells for future research on alveolar lung diseases.
Identifiants
pubmed: 30773483
pii: S2213-6711(19)30016-5
doi: 10.1016/j.stemcr.2019.01.014
pmc: PMC6409438
pii:
doi:
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
431-440Commentaires et corrections
Type : ErratumIn
Informations de copyright
Copyright © 2019 The Author(s). Published by Elsevier Inc. All rights reserved.
Références
Cell Stem Cell. 2017 Oct 5;21(4):472-488.e10
pubmed: 28965766
Pediatr Res. 2002 Feb;51(2):150-8
pubmed: 11809908
J Histochem Cytochem. 2010 Oct;58(10):891-901
pubmed: 20566753
Clin Chest Med. 2016 Sep;37(3):505-11
pubmed: 27514596
Nat Immunol. 2003 Nov;4(11):1111-20
pubmed: 14566336
Mol Biol Cell. 2002 Mar;13(3):1071-82
pubmed: 11907283
Nat Methods. 2017 Nov;14(11):1097-1106
pubmed: 28967890
J Cell Sci. 1993 Sep;106 ( Pt 1):99-107
pubmed: 8270647
Blood. 1999 Jul 1;94(1):146-55
pubmed: 10381507
Cancer Immun. 2008 Feb 06;8:3
pubmed: 18251464
Am J Physiol Lung Cell Mol Physiol. 2003 Sep;285(3):L643-53
pubmed: 12777251
Virchows Arch. 2000 Sep;437(3):304-13
pubmed: 11037352
Nat Cell Biol. 2017 May;19(5):542-549
pubmed: 28436965
Am J Respir Cell Mol Biol. 2005 Jul;33(1):14-21
pubmed: 15790974
FEBS Lett. 2001 Nov 16;508(2):221-5
pubmed: 11718719
Stem Cell Reports. 2016 Jan 12;6(1):18-25
pubmed: 26724905
Am J Respir Cell Mol Biol. 2007 Jul;37(1):67-74
pubmed: 17363777
Genetics. 1979 May;92(1):189-204
pubmed: 115747
Mol Cell. 1999 Jan;3(1):11-21
pubmed: 10024875
Stem Cell Reports. 2014 Sep 9;3(3):394-403
pubmed: 25241738
Am J Respir Cell Mol Biol. 2018 May;58(5):566-574
pubmed: 29190429
Annu Rev Pathol. 2015;10:371-93
pubmed: 25621661
Am J Physiol Lung Cell Mol Physiol. 2002 Nov;283(5):L1079-85
pubmed: 12376361
J Clin Invest. 2013 Jul;123(7):3025-36
pubmed: 23921127
Proc Natl Acad Sci U S A. 1998 Feb 17;95(4):1579-84
pubmed: 9465058
J Clin Invest. 2017 Jun 1;127(6):2277-2294
pubmed: 28463226
Blood. 2013 Apr 11;121(15):2943-51
pubmed: 23403622
Stem Cell Reports. 2015 Jan 13;4(1):143-154
pubmed: 25434822