Use of PTC124 for nonsense suppression therapy targeting BMP4 nonsense variants in vitro and the bmp4^st72 allele in zebrafish

• Max Krall ,

  Contributed equally to this work with: Max Krall, Stephanie Htun

  Roles Formal analysis, Investigation, Methodology, Validation, Visualization, Writing – review & editing

  Affiliation Division of Genetics, Department of Pediatrics, University of California San Francisco, San Francisco, CA, United States of America

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• Stephanie Htun ,

  Contributed equally to this work with: Max Krall, Stephanie Htun

  Roles Conceptualization, Formal analysis, Investigation, Methodology, Validation, Visualization, Writing – review & editing

  Affiliation Division of Genetics, Department of Pediatrics, University of California San Francisco, San Francisco, CA, United States of America

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• Anne Slavotinek

  Roles Conceptualization, Formal analysis, Methodology, Project administration, Resources, Supervision, Writing – original draft

  * E-mail: anne.slavotinek@ucsf.edu

  Affiliation Division of Genetics, Department of Pediatrics, University of California San Francisco, San Francisco, CA, United States of America

  http://orcid.org/0000-0001-7053-2449

  ⨯