©The Author(s) 2017. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastroenterol. Aug 14, 2017; 23(30): 5451-5456
Published online Aug 14, 2017. doi: 10.3748/wjg.v23.i30.5451
Published online Aug 14, 2017. doi: 10.3748/wjg.v23.i30.5451
Wilson’s disease: Prospective developments towards new therapies
Giusy Ranucci, Raffaele Iorio, Department of Translational Medical Science, Section of Pediatric, University Federico II, 80131 Naples, Italy
Roman Polishchuck, Telethon Institute of Genetics and Medicine, 80078 Pozzuoli (NA), Italy
Author contributions: Ranucci G, Polishchuck R and Iorio R contributed equally to the article’s design, conception and writing.
Conflict-of-interest statement: All authors declare they have no conflicts of interest.
Correspondence to: Raffaele Iorio, MD, Associate Professor, Department of Translational Medical Science, Section of Pediatrics, University Federico II, Via Pansini 5, 80131 Naples, Italy. riorio@unina.it
Telephone: +39-081-7464337 Fax: +39-081-7464337
Received: February 3, 2017
Peer-review started: February 8, 2017
First decision: March 6, 2017
Revised: April 11, 2017
Accepted: July 22, 2017
Article in press: July 24, 2017
Published online: August 14, 2017
Processing time: 191 Days and 6.4 Hours
Peer-review started: February 8, 2017
First decision: March 6, 2017
Revised: April 11, 2017
Accepted: July 22, 2017
Article in press: July 24, 2017
Published online: August 14, 2017
Processing time: 191 Days and 6.4 Hours
Core Tip
Core tip: Hepatocytes derived from human-induced pluripotent stem cells hold great promise for the drug discovery process, especially for Wilson’s disease (WD). Therapeutic approaches offering correction of the ATP7B mutant function and/or less toxic suppression of copper accumulation are promising and could be available shortly. In particular, protein quality control components and their regulatory networks represent attractive new targets for WD-causing mutant correction. Cell and gene therapies, however, will require more studies before they can be considered for clinical trials. A key goal for WD advancement is international cooperation of specialized centers to overcome limited availability of patients for study.