Application of CRISPR/Cas9 editing and digital droplet PCR in human iPSCs to generate novel knock-in reporter lines to visualize dopaminergic neurons.


Journal

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

Informations de publication

Date de publication:
12 2019
Historique:
received: 19 12 2018
revised: 30 10 2019
accepted: 08 11 2019
pubmed: 17 11 2019
medline: 3 6 2020
entrez: 17 11 2019
Statut: ppublish

Résumé

Human induced pluripotent stem cells (hiPSCs) have become indispensable for disease modelling. They are an important resource to access patient cells harbouring disease-causing mutations. Derivation of midbrain dopaminergic (DAergic) neurons from hiPSCs of PD patients represents the only option to model physiological processes in a cell type that is not otherwise accessible from human patients. However, differentiation does not produce a homogenous population of DA neurons and contaminant cell types may interfere with the readout of the in vitro system. Here, we use CRISPR/Cas9 to generate novel knock-in reporter lines for DA neurons, engineered with an endogenous fluorescent tyrosine hydroxylase - enhanced green fluorescent protein (TH-eGFP) reporter. We present a reproducible knock-in strategy combined with a highly specific homologous directed repair (HDR) screening approach using digital droplet PCR (ddPCR). The knock-in cell lines that we created show a functioning fluorescent reporter system for DA neurons that are identifiable by flow cytometry.

Identifiants

pubmed: 31733438
pii: S1873-5061(19)30286-7
doi: 10.1016/j.scr.2019.101656
pmc: PMC7322529
pii:
doi:

Substances chimiques

enhanced green fluorescent protein 0
Green Fluorescent Proteins 147336-22-9

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

101656

Subventions

Organisme : Medical Research Council
ID : MR/L023784/1
Pays : United Kingdom
Organisme : Medical Research Council
ID : MC_EX_MR/N50192X/1
Pays : United Kingdom
Organisme : Medical Research Council
ID : MR/L023784/2
Pays : United Kingdom
Organisme : Medical Research Council
ID : MR/M024962/1
Pays : United Kingdom
Organisme : Wellcome Trust
ID : WTISSF121302
Pays : United Kingdom
Organisme : Parkinson's UK
ID : J-0901
Pays : United Kingdom

Informations de copyright

Copyright © 2019 The Authors. Published by Elsevier B.V. All rights reserved.

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Auteurs

Christa Überbacher (C)

Institute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, Italy; Department of Biomedical, Metabolic and Neural Sciences, Università di Modena e Reggio Emilia, Modena, Italy. Electronic address: christa.ueberbacher@i-med.ac.at.

Julia Obergasteiger (J)

Institute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, Italy.

Mattia Volta (M)

Institute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, Italy.

Serena Venezia (S)

Institute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, Italy.

Stefan Müller (S)

Institute of Human Genetics, Munich University Hospital, Ludwig-Maximilians University, Munich, Germany.

Isabella Pesce (I)

CIBIO - Centre for Integrative Biology, Università degli Studi di Trento, Trento, Italy.

Sara Pizzi (S)

Institute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, Italy.

Giulia Lamonaca (G)

Institute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, Italy.

Anne Picard (A)

Institute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, Italy.

Giada Cattelan (G)

Institute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, Italy.

Giorgio Malpeli (G)

Department of Surgical Sciences, Dentistry, Gynecology and Pediatrics, Section of Surgery, University of Verona, Verona, Italy; Department of Diagnostics and Public Health, University of Verona, Verona, Italy.

Michele Zoli (M)

Department of Biomedical, Metabolic and Neural Sciences, Università di Modena e Reggio Emilia, Modena, Italy; Center for Neuroscience and Neurotechnology, University of Modena and Reggio Emilia, Modena, Italy.

Dayne Beccano-Kelly (D)

Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK; Oxford Parkinson's Disease Centre, University of Oxford, Oxford, UK.

Rowan Flynn (R)

James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Oxford, UK.

Richard Wade-Martins (R)

Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK; Oxford Parkinson's Disease Centre, University of Oxford, Oxford, UK.

Peter P Pramstaller (PP)

Institute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, Italy.

Andrew A Hicks (AA)

Institute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, Italy.

Sally A Cowley (SA)

Oxford Parkinson's Disease Centre, University of Oxford, Oxford, UK; James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Oxford, UK.

Corrado Corti (C)

Institute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, Italy. Electronic address: corrado.corti@eurac.edu.

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