Evaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells

Christoph J. Niemietz; Vanessa Sauer; Jacqueline Stella; Lutz Fleischhauer; Gursimran Chandhok; Sarah Guttmann; Yesim Avsar; Shuling Guo; Elizabeth J. Ackermann; Jared Gollob; Brett P. Monia; Andree Zibert; Hartmut H. -J. Schmidt

doi:10.1371/journal.pone.0161455

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Open Access

Peer-reviewed

Research Article

Evaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells

Christoph J. Niemietz,

Affiliation Klinik für Transplantationsmedizin, Universitätsklinikum Münster, Albert-Schweitzer-Campus 1, Gebäude A14, D-48149, Münster, Germany
⨯
Vanessa Sauer,

Affiliation Klinik für Transplantationsmedizin, Universitätsklinikum Münster, Albert-Schweitzer-Campus 1, Gebäude A14, D-48149, Münster, Germany
⨯
Jacqueline Stella,

Affiliation Klinik für Transplantationsmedizin, Universitätsklinikum Münster, Albert-Schweitzer-Campus 1, Gebäude A14, D-48149, Münster, Germany
⨯
Lutz Fleischhauer,

Affiliation Klinik für Transplantationsmedizin, Universitätsklinikum Münster, Albert-Schweitzer-Campus 1, Gebäude A14, D-48149, Münster, Germany
⨯
Gursimran Chandhok,

Affiliation Klinik für Transplantationsmedizin, Universitätsklinikum Münster, Albert-Schweitzer-Campus 1, Gebäude A14, D-48149, Münster, Germany
⨯
Sarah Guttmann,

Affiliation Klinik für Transplantationsmedizin, Universitätsklinikum Münster, Albert-Schweitzer-Campus 1, Gebäude A14, D-48149, Münster, Germany
⨯
Yesim Avsar,

Affiliation Klinik für Transplantationsmedizin, Universitätsklinikum Münster, Albert-Schweitzer-Campus 1, Gebäude A14, D-48149, Münster, Germany
⨯
Shuling Guo,

Affiliation Ionis Pharmaceuticals, Inc., 2855 Gazelle Ct, Carlsbad, CA, 92010, United States of America
⨯
Elizabeth J. Ackermann,

Affiliation Ionis Pharmaceuticals, Inc., 2855 Gazelle Ct, Carlsbad, CA, 92010, United States of America
⨯
Jared Gollob,

Affiliation Alnylam Pharmaceuticals, Inc., 300 Third St #3, Cambridge, MA, 02142, United States of America
⨯
Brett P. Monia,

Affiliation Ionis Pharmaceuticals, Inc., 2855 Gazelle Ct, Carlsbad, CA, 92010, United States of America
⨯
Andree Zibert,

Affiliation Klinik für Transplantationsmedizin, Universitätsklinikum Münster, Albert-Schweitzer-Campus 1, Gebäude A14, D-48149, Münster, Germany
⨯
Hartmut H. -J. Schmidt

* E-mail: hepar@ukmuenster.de

Affiliation Klinik für Transplantationsmedizin, Universitätsklinikum Münster, Albert-Schweitzer-Campus 1, Gebäude A14, D-48149, Münster, Germany
⨯

Evaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells

Christoph J. Niemietz,
Vanessa Sauer,
Jacqueline Stella,
Lutz Fleischhauer,
Gursimran Chandhok,
Sarah Guttmann,
Yesim Avsar,
Shuling Guo,
Elizabeth J. Ackermann,
Jared Gollob

Published: September 1, 2016
https://doi.org/10.1371/journal.pone.0161455

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