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Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery

Human-induced pluripotent stem cells (hiPSCs) and CRISPR/Cas9 gene editing system represent two instruments of basic and translational research, which both allow to acquire deep insight about the molecular bases of many diseases but also to develop pharmacological research. This review is focused to...

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Autores principales: De Masi, Claudia, Spitalieri, Paola, Murdocca, Michela, Novelli, Giuseppe, Sangiuolo, Federica
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7318728/
https://www.ncbi.nlm.nih.gov/pubmed/32591003
http://dx.doi.org/10.1186/s40246-020-00276-2
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author De Masi, Claudia
Spitalieri, Paola
Murdocca, Michela
Novelli, Giuseppe
Sangiuolo, Federica
author_facet De Masi, Claudia
Spitalieri, Paola
Murdocca, Michela
Novelli, Giuseppe
Sangiuolo, Federica
author_sort De Masi, Claudia
collection PubMed
description Human-induced pluripotent stem cells (hiPSCs) and CRISPR/Cas9 gene editing system represent two instruments of basic and translational research, which both allow to acquire deep insight about the molecular bases of many diseases but also to develop pharmacological research. This review is focused to draw up the latest technique of gene editing applied on hiPSCs, exploiting some of the genetic manipulation directed to the discovery of innovative therapeutic strategies. There are many expediencies provided by the use of hiPSCs, which can represent a disease model clinically relevant and predictive, with a great potential if associated to CRISPR/Cas9 technology, a gene editing tool powered by ease and precision never seen before. Here, we describe the possible applications of CRISPR/Cas9 to hiPSCs: from drug development to drug screening and from gene therapy to the induction of the immunological response to specific virus infection, such as HIV and SARS-Cov-2.
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spelling pubmed-73187282020-06-29 Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery De Masi, Claudia Spitalieri, Paola Murdocca, Michela Novelli, Giuseppe Sangiuolo, Federica Hum Genomics Review Human-induced pluripotent stem cells (hiPSCs) and CRISPR/Cas9 gene editing system represent two instruments of basic and translational research, which both allow to acquire deep insight about the molecular bases of many diseases but also to develop pharmacological research. This review is focused to draw up the latest technique of gene editing applied on hiPSCs, exploiting some of the genetic manipulation directed to the discovery of innovative therapeutic strategies. There are many expediencies provided by the use of hiPSCs, which can represent a disease model clinically relevant and predictive, with a great potential if associated to CRISPR/Cas9 technology, a gene editing tool powered by ease and precision never seen before. Here, we describe the possible applications of CRISPR/Cas9 to hiPSCs: from drug development to drug screening and from gene therapy to the induction of the immunological response to specific virus infection, such as HIV and SARS-Cov-2. BioMed Central 2020-06-26 /pmc/articles/PMC7318728/ /pubmed/32591003 http://dx.doi.org/10.1186/s40246-020-00276-2 Text en © The Author(s) 2020 Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Review
De Masi, Claudia
Spitalieri, Paola
Murdocca, Michela
Novelli, Giuseppe
Sangiuolo, Federica
Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery
title Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery
title_full Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery
title_fullStr Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery
title_full_unstemmed Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery
title_short Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery
title_sort application of crispr/cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7318728/
https://www.ncbi.nlm.nih.gov/pubmed/32591003
http://dx.doi.org/10.1186/s40246-020-00276-2
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