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Copyright ©The Author(s) 2016. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Stem Cells. Apr 26, 2016; 8(4): 118-135
Published online Apr 26, 2016. doi: 10.4252/wjsc.v8.i4.118
Human induced pluripotent stem cells for monogenic disease modelling and therapy
Paola Spitalieri, Valentina Rosa Talarico, Michela Murdocca, Giuseppe Novelli, Federica Sangiuolo
Paola Spitalieri, Valentina Rosa Talarico, Michela Murdocca, Giuseppe Novelli, Federica Sangiuolo, Department of Biomedicine and Prevention, Tor Vergata University, 00133 Rome, Italy
Author contributions: Spitalieri P and Sangiuolo F contributed to drafting and making critical revisions of the article; Talarico VR and Murdocca M contributed to analysis and interpretation of data; Novelli G contributed to final approval of the version of the article to be published.
Supported by Agenzia Spaziale Italiana (ASI), CoReA, No. 2013-084-R.0.
Conflict-of-interest statement: The authors declare no conflicts of interests for this article.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Correspondence to: Federica Sangiuolo, PhD, Department of Biomedicine and Prevention, Tor Vergata University, via Montpellier, 1, 00133 Rome, Italy. sangiuolo@med.uniroma2.it
Telephone: +39-06-72596164 Fax: +39-06-20427313
Received: September 9, 2015
Peer-review started: September 10, 2015
First decision: October 8, 2015
Revised: January 21, 2016
Accepted: February 14, 2016
Article in press: February 16, 2016
Published online: April 26, 2016
Processing time: 216 Days and 19.5 Hours
Core Tip

Core tip: With the development of human induced pluripotent stem cells (hiPSCs) deriving from patients, we can begin to understand the molecular mechanisms underlying monogenic diseases and consequently identify new drugs for their treatment. hiPSCs can differentiate into many disease-relevant cell types, providing in this way to innovative applications in the field of cell replacement therapy, disease modelling, drug testing and drug discovery.