About the Authors

Carla Casu

Roles Data curation, Writing – original draft

casu.carla@gmail.com

Affiliation Division of Hematology, Department of Pediatrics, The Children’s Hospital of Philadelphia (CHOP), Philadelphia, PA, United States of America

Alison Liu

Roles Data curation

Affiliation Division of Hematology, Department of Pediatrics, The Children’s Hospital of Philadelphia (CHOP), Philadelphia, PA, United States of America

Gianluca De Rosa

Roles Data curation

Affiliation Division of Hematology, Department of Pediatrics, The Children’s Hospital of Philadelphia (CHOP), Philadelphia, PA, United States of America

Audrey Low

Roles Data curation, Formal analysis

Affiliation Ionis Pharmaceuticals, Inc., Carlsbad, CA, United States of America

Aae Suzuki

Roles Data curation

Affiliation University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, United States of America

Sayantani Sinha

Roles Data curation

Affiliation Division of Hematology, Department of Pediatrics, The Children’s Hospital of Philadelphia (CHOP), Philadelphia, PA, United States of America

Yelena Z. Ginzburg

Roles Formal analysis, Writing – review & editing

Affiliation Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, United States of America

Charles Abrams

Roles Formal analysis, Writing – review & editing

Affiliation University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, United States of America

Mariam Aghajan

Roles Resources

Affiliation Ionis Pharmaceuticals, Inc., Carlsbad, CA, United States of America

Shuling Guo

Roles Resources

Affiliation Ionis Pharmaceuticals, Inc., Carlsbad, CA, United States of America

Stefano Rivella

Roles Writing – original draft, Writing – review & editing

Affiliations Division of Hematology, Department of Pediatrics, The Children’s Hospital of Philadelphia (CHOP), Philadelphia, PA, United States of America, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, United States of America, Cell and Molecular Biology affinity group (CAMB), University of Pennsylvania, Philadelphia, PA, United States of America, Raymond G. Perelman Center for Cellular and Molecular Therapeutics-CHOP, Philadelphia, PA, United States of America, Penn Center for Musculoskeletal Disorders, CHOP, Philadelphia, PA, United States of America

Competing Interests

S.R. is a member of scientific advisory board of Ionis Pharmaceuticals, Meira GTx and Disc Medicine and owns stock options from Disc Medicine and Meira GTx. S.R. has been or is consultant for Cambridge Healthcare Res, Celgene Corporation, Ghost Tree Capital, Noble insight, Protagonist Therapeutics, Sanofi Aventis U.S., Inc, Slingshot Insight, Techspert.io and venBio Select LLC. and Disc Medicine. S.G. M.A. and A. L. are employees and shareholders of Ionis Pharmaceuticals. Y.Z.G. is a consultant for Ionis Pharmaceuticals and Protagonist Therapeutics and receives research funding from Protagonist Therapeutics. The remaining authors declare no competing financial interests. Ionis Pharmaceuticals did not provide financial support to this study and only had roles in the study design, data collection and analysis, decision to publish, and preparation of the manuscript. Participation of Ionis Pharmaceuticals to the study does not alter our adherence to PLOS ONE policies on sharing data and materials.