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

Binyamin Eisen, Ronen Ben Jehuda, Ashley J. Cuttitta, Lucy N. Mekies, Irina Reiter, Sindhu Ramchandren, Michael Arad, Daniel E. Michele, Ofer Binah*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

Abstract

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.

Original languageEnglish
Pages (from-to)111-114
Number of pages4
JournalStem Cell Research
Volume29
DOIs
StatePublished - May 2018

Funding

FundersFunder number
National Institute of Arthritis and Musculoskeletal and Skin DiseasesR01AR068428

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