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Generation of genomic-integration-free human induced pluripotent stem cells and the derived cardiomyocytes of X-linked dilated cardiomyopathy from DMD gene mutation.
Zhu, Sheng; Law, Anna Hing Yee; Deng, Ruixia; Poon, Ellen Ngar Yun; Lo, Chun Wai; Kwong, Anna Ka Yee; Liang, Rui; Chan, Kelvin Yuen Kwong; Wong, Wai Lap; Tan-Un, Kian Cheng; Pijnappel, W W M Pim; Chan, Godfrey Chi Fung; Chan, Sophelia Hoi Shan.
Afiliação
  • Zhu S; Department of Paediatrics and Adolescent Medicine, The University of Hong Kong, Queen Mary Hospital, Hong Kong, HKSAR. Electronic address: zhusheng@connect.hku.hk.
  • Law AHY; Department of Paediatrics and Adolescent Medicine, The University of Hong Kong, Queen Mary Hospital, Hong Kong, HKSAR. Electronic address: lawanna@hku.hk.
  • Deng R; Department of Paediatrics and Adolescent Medicine, The University of Hong Kong, Queen Mary Hospital, Hong Kong, HKSAR. Electronic address: margaretrx.deng@gmail.com.
  • Poon ENY; Centre for Cardiovascular Genomics and Medicine, Lui Che Woo Institute of Innovative Medicine, Hong Kong, HKSAR; Hong Kong Hub of Paediatric Excellence (HK HOPE), Hong Kong, HKSAR; Department of Medicine and Therapeutics, The Chinese University of Hong Kong, Hong Kong, HKSAR. Electronic address: ell
  • Lo CW; Department of Paediatrics and Adolescent Medicine, The University of Hong Kong, Queen Mary Hospital, Hong Kong, HKSAR. Electronic address: jo94490@hku.hk.
  • Kwong AKY; Department of Paediatrics and Adolescent Medicine, The University of Hong Kong, Queen Mary Hospital, Hong Kong, HKSAR. Electronic address: kkyanna@hku.hk.
  • Liang R; Department of Paediatrics and Adolescent Medicine, The University of Hong Kong, Queen Mary Hospital, Hong Kong, HKSAR. Electronic address: liangrui@connect.hku.hk.
  • Chan KYK; Department of Obstetrics and Gynaecology, Queen Mary Hospital, Hong Kong, HKSAR; Prenatal Diagnostic Laboratory, Tsan Yuk Hospital, Hong Kong, HKSAR. Electronic address: ykchanc@hku.hk.
  • Wong WL; Department of Obstetrics and Gynaecology, Queen Mary Hospital, Hong Kong, HKSAR. Electronic address: wlapwong@hku.hk.
  • Tan-Un KC; School of Biological Science, The University of Hong Kong, Hong Kong, HKSAR. Electronic address: kctanun@hku.hk.
  • Pijnappel WWMP; Clinical Genetics and Pediatrics, Erasmus MC University, Medical Centre, Rotterdam, Netherlands. Electronic address: w.pijnappel@erasmusmc.nl.
  • Chan GCF; Department of Paediatrics and Adolescent Medicine, The University of Hong Kong, Queen Mary Hospital, Hong Kong, HKSAR. Electronic address: gcfchan@hku.hk.
  • Chan SHS; Department of Paediatrics and Adolescent Medicine, The University of Hong Kong, Queen Mary Hospital, Hong Kong, HKSAR. Electronic address: sophehs@hku.hk.
Stem Cell Res ; 49: 102040, 2020 12.
Article em En | MEDLINE | ID: mdl-33099108
We derived an integration-free induced pluripotent stem cell (iPSC) line from the peripheral blood mononuclear cells (PBMCs) of a 23-year-old male patient. This patient carries a 5' splice site point mutation in intron 1 (c.31+1G>A) of the dystrophin gene, a mutation associated with X-linked dilated cardiomyopathy (XLDCM). Sendai virus was used to reprogram the PBMCs and deliver OCT3/4, SOX2, c-MYC, and KLF4 factors. The iPSC line (HKUi002-A) generated preserved the mutation, expressed common pluripotency markers, differentiated into three germ layers in vivo, and exhibited a normal karyotype. Further differentiation into cardiomyocytes enables the study of the disease mechanisms of XLDCM.
Assuntos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Células-Tronco Pluripotentes Induzidas Limite: Adult / Humans / Male Idioma: En Revista: Stem Cell Res Ano de publicação: 2020 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Células-Tronco Pluripotentes Induzidas Limite: Adult / Humans / Male Idioma: En Revista: Stem Cell Res Ano de publicação: 2020 Tipo de documento: Article