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

CRISPR/Cas9 Drug discovery Gene editing Gene therapy HIV and SARS-Cov-2 infection Human-induced pluripotent stem cells (hiPSCs)

Journal

Human genomics
ISSN: 1479-7364
Titre abrégé: Hum Genomics
Pays: England
ID NLM: 101202210

Informations de publication

Date de publication:
26 06 2020
Historique:
received: 07 05 2020
accepted: 17 06 2020
entrez: 28 6 2020
pubmed: 28 6 2020
medline: 14 7 2020
Statut: epublish

Résumé

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.

Identifiants

pubmed: 32591003
doi: 10.1186/s40246-020-00276-2
pii: 10.1186/s40246-020-00276-2
pmc: PMC7318728
doi:

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

25

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Auteurs

Claudia De Masi (C)

Department of Biomedicine and Prevention, University of Rome "Tor Vergata", Rome, Italy.

Paola Spitalieri (P)

Department of Biomedicine and Prevention, University of Rome "Tor Vergata", Rome, Italy.

Michela Murdocca (M)

Department of Biomedicine and Prevention, University of Rome "Tor Vergata", Rome, Italy.

Giuseppe Novelli (G)

Department of Biomedicine and Prevention, University of Rome "Tor Vergata", Rome, Italy.

Federica Sangiuolo (F)

Department of Biomedicine and Prevention, University of Rome "Tor Vergata", Rome, Italy. sangiuolo@med.uniroma2.it.

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