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.

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, 14(1), 25 [10.1186/s40246-020-00276-2].

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

Spitalieri, P;Murdocca, M;Novelli, G;Sangiuolo, F
2020-01-01

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.
2020
Pubblicato
Rilevanza internazionale
Articolo
Esperti anonimi
Settore MED/03 - GENETICA MEDICA
English
Human-induced pluripotent stem cells (hiPSCs)
CRISPR
Cas9
Gene editing
Drug discovery
HIV and SARS-Cov-2 infection
Gene therapy
Animals
Cellular Reprogramming
Humans
Induced Pluripotent Stem Cells
Virus Diseases
CRISPR-Cas Systems
Drug Discovery
Gene Editing
Genetic Therapy
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, 14(1), 25 [10.1186/s40246-020-00276-2].
De Masi, C; Spitalieri, P; Murdocca, M; Novelli, G; Sangiuolo, F
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2108/256551
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