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Open Access
Peer-reviewed
Research Article
Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B
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Simon Krooss,
Roles Investigation, Writing – original draft
Affiliations Institute of Virology, Hannover Medical School, Hannover, Germany, Department of Gastroenterology, Hepatology and Endocrinology, Hannover Medical School and Twincore Centre for Experimental and Clinical Infection Research, Hannover, Germany
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Sonja Werwitzke,
Roles Formal analysis, Resources, Writing – review & editing
Affiliation Clinic of Hematology, Oncology and Hemostaseology, Hannover Medical School, Hannover, Germany
⨯ - Johannes Kopp,
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Alice Rovai,
Roles Methodology
Affiliation Department of Gastroenterology, Hepatology and Endocrinology, Hannover Medical School and Twincore Centre for Experimental and Clinical Infection Research, Hannover, Germany
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Dirk Varnholt,
Roles Formal analysis, Methodology, Writing – review & editing
Affiliation Clinic of Hematology, Oncology and Hemostaseology, Hannover Medical School, Hannover, Germany
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Amelie S. Wachs,
Roles Formal analysis, Resources, Writing – review & editing
Affiliation Institute of Virology, Hannover Medical School, Hannover, Germany
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Aurelie Goyenvalle,
Roles Resources, Writing – review & editing
Affiliation Université de Versailles St-Quentin en Yvelines, INSERM U1179, France
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Annemieke Aarstma-Rus,
Roles Resources, Writing – review & editing
Affiliation Leiden University Medical Center, Leiden, Netherlands
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Michael Ott,
Roles Resources, Writing – review & editing
Affiliation Department of Gastroenterology, Hepatology and Endocrinology, Hannover Medical School and Twincore Centre for Experimental and Clinical Infection Research, Hannover, Germany
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Andreas Tiede,
Roles Conceptualization, Methodology, Resources, Writing – review & editing
Affiliation Clinic of Hematology, Oncology and Hemostaseology, Hannover Medical School, Hannover, Germany
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Jörg Langemeier ,
Roles Conceptualization, Investigation, Methodology, Supervision, Writing – original draft, Writing – review & editing
* E-mail: joerglangemeier@web.de (JL); bohne.jens@mh-hannover.de (JB)
Affiliations Institute of Virology, Hannover Medical School, Hannover, Germany, Pediatric Intensive Care Unit, Children’s Hospital Bielefeld, Germany
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Jens Bohne
Roles Conceptualization, Funding acquisition, Investigation, Project administration, Resources, Supervision, Validation, Visualization, Writing – original draft, Writing – review & editing
* E-mail: joerglangemeier@web.de (JL); bohne.jens@mh-hannover.de (JB)
Affiliation Institute of Virology, Hannover Medical School, Hannover, Germany
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Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B
- Simon Krooss,
- Sonja Werwitzke,
- Johannes Kopp,
- Alice Rovai,
- Dirk Varnholt,
- Amelie S. Wachs,
- Aurelie Goyenvalle,
- Annemieke Aarstma-Rus,
- Michael Ott,
- Andreas Tiede
- Published: April 8, 2020
- https://doi.org/10.1371/journal.pgen.1008690