Generation of Duchenne muscular dystrophy patient-specific induced pluripotent stem cell line lacking exons 45-50 of the dystrophin gene (IITi001-A).

Autor: Eisen B; Department of Physiology, Biophysics and Systems Biology, Ruth and Bruce Rappaport Faculty of Medicine, Technion - Israel Institute of Technology, Haifa, Israel., Ben Jehuda R; Department of Physiology, Biophysics and Systems Biology, Ruth and Bruce Rappaport Faculty of Medicine, Technion - Israel Institute of Technology, Haifa, Israel; Department of Biotechnology, Technion - Israel Institute of Technology, Haifa, Israel., Cuttitta AJ; Department of Molecular and Integrative Physiology, University of Michigan Medical School, Ann Arbor, MI, USA., Mekies LN; Department of Physiology, Biophysics and Systems Biology, Ruth and Bruce Rappaport Faculty of Medicine, Technion - Israel Institute of Technology, Haifa, Israel., Reiter I; Department of Physiology, Biophysics and Systems Biology, Ruth and Bruce Rappaport Faculty of Medicine, Technion - Israel Institute of Technology, Haifa, Israel., Ramchandren S; Department of Neurology, University of Michigan Medical School, Ann Arbor, MI, USA., Arad M; Leviev Heart Center, Sheba Medical Center, Ramat Gan, Israel; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel., Michele DE; Department of Molecular and Integrative Physiology, University of Michigan Medical School, Ann Arbor, MI, USA. Electronic address: dmichele@umich.edu., Binah O; Department of Physiology, Biophysics and Systems Biology, Ruth and Bruce Rappaport Faculty of Medicine, Technion - Israel Institute of Technology, Haifa, Israel. Electronic address: binah@tx.technion.ac.il.
Jazyk: angličtina
Zdroj: Stem cell research [Stem Cell Res] 2018 May; Vol. 29, pp. 111-114. Date of Electronic Publication: 2018 Apr 03.
DOI: 10.1016/j.scr.2018.03.023
Abstrakt: Duchenne muscular dystrophy (DMD) is an X-linked progressive muscle degenerative disease caused by mutations in the dystrophin gene. We generated induced pluripotent stem cells (iPSCs) from a 13-year-old male patient carrying a deletion mutation of exons 45-50; iPSCs were subsequently differentiated into cardiomyocytes. iPSCs exhibit expression of the pluripotent markers (SOX2, NANOG, OCT4), differentiation capacity into the three germ layers, normal karyotype, genetic identity to the skin biopsy dermal fibroblasts and the patient-specific dystrophin mutation.
(Copyright © 2018 The Authors. Published by Elsevier B.V. All rights reserved.)
Databáze: MEDLINE