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Production and characterization of CSSI003 (2961) human induced pluripotent stem cells (iPSCs) carrying a novel puntiform mutation in RAI1 gene, Causative of Smith–Magenis syndrome

Authors :
Filomena Altieri
Elisa Maria Turco
Ersilia Vinci
Barbara Torres
Daniela Ferrari
Antonella De Jaco
Gianluigi Mazzoccoli
Giuseppe Lamorte
Annamaria Nardone
Matteo Della Monica
Laura Bernardini
Angelo Luigi Vescovi
Jessica Rosati
Source :
Stem Cell Research, Vol 28, Iss , Pp 153-156 (2018)
Publication Year :
2018
Publisher :
Elsevier, 2018.

Abstract

Smith-Magenis syndrome (SMS) is a complex genetic disorder characterized by developmental delay, behavioural problems and circadian rhythm dysregulation. About 90% of SMS cases are due to a 17p11.2 deletion containing retinoic acid induced1 (RAI1) gene, 10% are due to heterozygous mutations affecting RAI1 coding region. Little is known about RAI1 role.

Subjects

Subjects :
Biology (General)
QH301-705.5

Details

Language :
English
ISSN :
18735061
Volume :
28
Issue :
153-156
Database :
Directory of Open Access Journals
Journal :
Stem Cell Research
Publication Type :
Academic Journal
Accession number :
edsdoj.404a5a3c638b401ebbc749f36ad597b3
Document Type :
article
Full Text :
https://doi.org/10.1016/j.scr.2018.02.016