Generation of a CFTR knock-in reporter cell line (MHHi006-A-1) from a human induced pluripotent stem cell line

Publication date: Available online 20 August 2019Source: Stem Cell ResearchAuthor(s): Lena Engels, Ruth Olmer, Jeanne de la Roche, Gudrun Göhring, Saskia Ulrich, Ralf Haller, Ulrich Martin, Sylvia MerkertAbstractCFTR encodes for a chloride ion channel expressed primarily in secretory epithelia in the airways, intestine, liver and other tissues. Mutations in the CFTR gene have been identified in people suffering from Cystic Fibrosis. Here, we established a CFTR knock-in reporter cell line from a human iPSC line (MHHi006-A) using TALEN technology. The reporter enables the monitoring and optimization of the differentiation of pluripotent stem cells into CFTR expressing epithelia on a single cell level, as well as the enrichment of CFTR positive cells, which represent an excellent tool for Cystic Fibrosis disease modelling, drug screening and ultimately cellular therapies.
Source: Stem Cell Research - Category: Stem Cells Source Type: research

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Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
Recent advances in adult stem cell biology have resulted in the development of organoid culture technologies using a variety of tissue sources such as intestine, lung and kidney [1]. Organoids are three-dimensional, multicellular structures that recapitulate tissue features of the parental organ and are usually grown from donor tissue fragments [1]. As organoids are functional expressions of individual genomes, these cultures are particularly useful to understand how genetic factors contribute to individual disease. As such, they are used to study hereditary diseases such as cystic fibrosis (CF), and more common diseases s...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: ERJ Methods Source Type: research
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Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
Abstract Until recently, major advances in drug development have been hampered by a lack of proper cell and tissue models; but the introduction of organoid technology has revolutionized this field. At the level of the gastrointestinal tract, the so-called mini-gut comprises all major cell types of native intestine and recapitulates the composition and function of native intestinal epithelium. The mini-gut can be classified as an intestinal organoid (IO), derived from pluripotent stem cells, or as an enteroid, consisting only of epithelial cells and generated from adult stem cells. Both classifications have been us...
Source: Drug Discovery Today - Category: Drugs & Pharmacology Authors: Tags: Drug Discov Today Source Type: research
Abstract Cystic fibrosis (CF) is a life-shortening genetic disease caused by mutations of CFTR, the gene encoding cystic fibrosis transmembrane conductance regulator. Despite considerable progress in CF therapies, targeting specific CFTR genotypes based on small molecules has been hindered because of the substantial genetic heterogeneity of CFTR mutations in patients with CF, which is difficult to assess by animal models in vivo. There are broadly four classes (e.g., II, III, and IV) of CF genotypes that differentially respond to current CF drugs (e.g., VX-770 and VX-809). In this review, we shed light on the phar...
Source: Drug Discovery Today - Category: Drugs & Pharmacology Authors: Tags: Drug Discov Today Source Type: research
Contributors : Sylvia Merkert ; Madline Schubert ; Ruth Olmer ; Lena Engels ; Silke Radetzki ; Mieke Veltman ; Bob Scholte ; Janina Z öllner ; Nicoletta Pedemonte ; Luis Galietta ; Jens von Kries ; Ulrich MartinSeries Type : Expression profiling by arrayOrganism : Homo sapiensOrganotypic culture systems from disease-specific induced pluripotent stem cells (iPSCs) exhibit obvious advantages compared to immortalized cell lines and primary cell cultures but implementation of iPSC-based high throughput (HT) assays is still technically challenging. Here we demonstrate the development and conduction of an organotypic HT Cl-...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Expression profiling by array Homo sapiens Source Type: research
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Source: Stem Cell Reports - Category: Stem Cells Source Type: research
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Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Cosmeri Rizzato1, Javier Torres2, Elena Kasamatsu3, Margarita Camorlinga-Ponce2, Maria Mercedes Bravo4, Federico Canzian5 and Ikuko Kato6* 1Department of Translation Research and of New Technologies in Medicine and Surgery, University of Pisa, Pisa, Italy 2Unidad de Investigación en Enfermedades Infecciosas, Unidades Médicas de Alta Especialidad Pediatría, Instituto Mexicano del Seguro Social, Mexico City, Mexico 3Instituto de Investigaciones en Ciencias de la Salud, National University of Asunción, Asunción, Paraguay 4Grupo de Investigación en Biología del C&aacut...
Source: Frontiers in Microbiology - Category: Microbiology Source Type: research
Mark E. Gray1,2*, James Meehan2,3, Paul Sullivan4, Jamie R. K. Marland4, Stephen N. Greenhalgh1, Rachael Gregson1, Richard Eddie Clutton1, Carol Ward2, Chris Cousens5, David J. Griffiths5, Alan Murray4 and David Argyle1 1The Royal (Dick) School of Veterinary Studies and Roslin Institute, University of Edinburgh, Edinburgh, United Kingdom 2Cancer Research UK Edinburgh Centre and Division of Pathology Laboratories, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, United Kingdom 3School of Engineering and Physical Sciences, Institute of Sensors, Signals and Systems, Heriot-Watt Univer...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
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