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Deriving human ENS lineages for cell therapy and drug discovery in Hirschsprung disease

Author

Listed:
  • Faranak Fattahi

    (The Center for Stem Cell Biology
    Developmental Biology Program, Sloan-Kettering Institute for Cancer Research
    Weill Graduate School of Medical Sciences of Cornell University)

  • Julius A Steinbeck

    (The Center for Stem Cell Biology
    Developmental Biology Program, Sloan-Kettering Institute for Cancer Research)

  • Sonja Kriks

    (The Center for Stem Cell Biology
    Developmental Biology Program, Sloan-Kettering Institute for Cancer Research)

  • Jason Tchieu

    (The Center for Stem Cell Biology
    Developmental Biology Program, Sloan-Kettering Institute for Cancer Research)

  • Bastian Zimmer

    (The Center for Stem Cell Biology
    Developmental Biology Program, Sloan-Kettering Institute for Cancer Research)

  • Sarah Kishinevsky

    (The Center for Stem Cell Biology
    Developmental Biology Program, Sloan-Kettering Institute for Cancer Research
    Weill Graduate School of Medical Sciences of Cornell University)

  • Nadja Zeltner

    (The Center for Stem Cell Biology
    Developmental Biology Program, Sloan-Kettering Institute for Cancer Research)

  • Yvonne Mica

    (The Center for Stem Cell Biology
    Developmental Biology Program, Sloan-Kettering Institute for Cancer Research
    † Present address: Thermo Fisher Scientific, Waltham, Massachusetts 02451, USA.)

  • Wael El-Nachef

    (Molecular Pharmacology Program)

  • Huiyong Zhao

    (Molecular Pharmacology Program)

  • Elisa de Stanchina

    (Molecular Pharmacology Program)

  • Michael D. Gershon

    (Columbia University, College of Physicians and Surgeons)

  • Tracy C. Grikscheit

    (Children’s Hospital Los Angeles, Pediatric Surgery)

  • Shuibing Chen

    (Weill Medical College of Cornell University)

  • Lorenz Studer

    (The Center for Stem Cell Biology
    Developmental Biology Program, Sloan-Kettering Institute for Cancer Research)

Abstract

A differentiation protocol to obtain enteric nervous system (ENS) progenitors and a range of neurons from human pluripotent stem cells is developed; the cells can migrate and graft to the colon of a chick embryo and an adult mouse colon, including in a mouse model of Hirschsprung disease, in which a functional rescue is observed.

Suggested Citation

  • Faranak Fattahi & Julius A Steinbeck & Sonja Kriks & Jason Tchieu & Bastian Zimmer & Sarah Kishinevsky & Nadja Zeltner & Yvonne Mica & Wael El-Nachef & Huiyong Zhao & Elisa de Stanchina & Michael D. G, 2016. "Deriving human ENS lineages for cell therapy and drug discovery in Hirschsprung disease," Nature, Nature, vol. 531(7592), pages 105-109, March.
    • Handle: RePEc:nat:nature:v:531:y:2016:i:7592:d:10.1038_nature16951
    DOI: 10.1038/nature16951
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    Cited by:

    1. Dianne Lumaquin-Yin & Emily Montal & Eleanor Johns & Arianna Baggiolini & Ting-Hsiang Huang & Yilun Ma & Charlotte LaPlante & Shruthy Suresh & Lorenz Studer & Richard M. White, 2023. "Lipid droplets are a metabolic vulnerability in melanoma," Nature Communications, Nature, vol. 14(1), pages 1-16, December.
    2. Weikang Pan & Ahmed A. Rahman & Takahiro Ohkura & Rhian Stavely & Kensuke Ohishi & Christopher Y. Han & Abigail Leavitt & Aki Kashiwagi & Alan J. Burns & Allan M. Goldstein & Ryo Hotta, 2024. "Autologous cell transplantation for treatment of colorectal aganglionosis in mice," Nature Communications, Nature, vol. 15(1), pages 1-10, December.
    3. Hsueh-Fu Wu & Wenxin Yu & Kenyi Saito-Diaz & Chia-Wei Huang & Joseph Carey & Frances Lefcort & Gerald W. Hart & Hong-Xiang Liu & Nadja Zeltner, 2022. "Norepinephrine transporter defects lead to sympathetic hyperactivity in Familial Dysautonomia models," Nature Communications, Nature, vol. 13(1), pages 1-17, December.

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