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Generation of familial amyloidotic polyneuropathy-specific induced pluripotent stem cells.
Isono, Kaori; Jono, Hirofumi; Ohya, Yuki; Shiraki, Nobuaki; Yamazoe, Taiji; Sugasaki, Ayaka; Era, Takumi; Fusaki, Noemi; Tasaki, Masayoshi; Ueda, Mitsuharu; Shinriki, Satoru; Inomata, Yukihiro; Kume, Shoen; Ando, Yukio.
Afiliação
  • Isono K; Department of Neurology, Graduate School of Medical Science, Kumamoto University, Kumamoto, Japan; Department of Transplantation and Pediatric Surgery, Graduate School of Medical Science, Kumamoto University, Kumamoto, Japan.
  • Jono H; Department of Diagnostic Medicine, Graduate School of Medical Science, Kumamoto University, Kumamoto, Japan; Department of Pharmacy, Kumamoto University Hospital, Kumamoto University, Kumamoto, Japan.
  • Ohya Y; Department of Transplantation and Pediatric Surgery, Graduate School of Medical Science, Kumamoto University, Kumamoto, Japan.
  • Shiraki N; Department of Stem Cell Biology, Institute of Molecular Embryology and Genetics, Kumamoto University, Kumamoto, Japan.
  • Yamazoe T; Department of Stem Cell Biology, Institute of Molecular Embryology and Genetics, Kumamoto University, Kumamoto, Japan.
  • Sugasaki A; Department of Neurology, Graduate School of Medical Science, Kumamoto University, Kumamoto, Japan.
  • Era T; Department of Cell Modulation, Institute of Molecular Embryology and Genetics, Kumamoto University, Kumamoto, Japan.
  • Fusaki N; DNAVEC Corporation, Ibaraki, Japan; Japan Science and Technology Agency (JST), PRESTO, Saitama, Japan; Ophthalmology, School of Medicine, Keio University, Japan.
  • Tasaki M; Department of Neurology, Graduate School of Medical Science, Kumamoto University, Kumamoto, Japan; Department of Diagnostic Medicine, Graduate School of Medical Science, Kumamoto University, Kumamoto, Japan.
  • Ueda M; Department of Diagnostic Medicine, Graduate School of Medical Science, Kumamoto University, Kumamoto, Japan.
  • Shinriki S; Department of Diagnostic Medicine, Graduate School of Medical Science, Kumamoto University, Kumamoto, Japan.
  • Inomata Y; Department of Transplantation and Pediatric Surgery, Graduate School of Medical Science, Kumamoto University, Kumamoto, Japan.
  • Kume S; Department of Stem Cell Biology, Institute of Molecular Embryology and Genetics, Kumamoto University, Kumamoto, Japan. Electronic address: skume@kumamoto-u.ac.jp.
  • Ando Y; Department of Neurology, Graduate School of Medical Science, Kumamoto University, Kumamoto, Japan; Department of Diagnostic Medicine, Graduate School of Medical Science, Kumamoto University, Kumamoto, Japan. Electronic address: andoy709@kumamoto-u.ac.jp.
Stem Cell Res ; 12(2): 574-83, 2014 Mar.
Article em En | MEDLINE | ID: mdl-24531302
ABSTRACT
Familial amyloidotic polyneuropathy (FAP) is a hereditary amyloidosis induced by amyloidogenic transthyretin (ATTR). Because most transthyretin (TTR) in serum is synthesized by the liver, liver transplantation (LT) is today the only treatment available to halt the progression of FAP, even though LT is associated with several problems. Despite the urgent need to develop alternatives to LT, the detailed pathogenesis of FAP is still unknown; also, no model fully represents the relevant processes in patients with FAP. The induction of induced pluripotent stem (iPS) cells has allowed development of pluripotent cells specific for patients and has led to useful models of human diseases. Because of the need for a tool to elucidate the molecular pathogenesis of FAP, in this study we sought to establish heterozygous ATTR mutant iPS cells, and were successful, by using a Sendai virus vector mixture containing four transcription factors (Oct3/4, Sox2, Klf4, and c-Myc) to reprogram dermal fibroblasts derived from FAP patients. Moreover, FAP-specific iPS cells had the potential to differentiate into hepatocyte-like cells and indeed expressed ATTR. FAP-specific iPS cells demonstrated the possibility of serving as a pathological tool that will contribute to understanding the pathogenesis of FAP and development of FAP treatments.
Assuntos

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Pré-Albumina / Neuropatias Amiloides Familiares / Células-Tronco Pluripotentes Induzidas Idioma: En Ano de publicação: 2014 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Pré-Albumina / Neuropatias Amiloides Familiares / Células-Tronco Pluripotentes Induzidas Idioma: En Ano de publicação: 2014 Tipo de documento: Article