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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·2020·Human Genomics
DOI 10.1186/s40246-020-00276-2PMC PMC7318728
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Abstract

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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Fig. 1 Workflow of the research involving hiPSCs and CRISPR/Cas9 gene editing for the investigation of new drugs and therapeutic alternatives
Fig. 1 Workflow of the research involving hiPSCs and CRISPR/Cas9 gene editing for the investigation of new drugs and therapeutic alternativesCC BY · PubMed Central

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Metadata

Authors
De Masi, C., Spitalieri, P., Murdocca, M., Novelli, G., & Sangiuolo, F.
ORCID
Sangiuolo, Federica
Venue
Human Genomics
Publisher
Springer Science and Business Media LLC
Published
2020-06-26
Volume
14
Issue
1
ISSN
1479-7364
DOI
10.1186/s40246-020-00276-2
PMC
PMC7318728
Cited by
91
References
13
Language
en
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CoChat. (2026). Citation check: Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery. https://app.cochat.ai/papers/10.1186/s40246-020-00276-2
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De Masi, C., Spitalieri, P., Murdocca, M., Novelli, G., & Sangiuolo, F. (2020). Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery. Human Genomics. https://doi.org/10.1186/s40246-020-00276-2