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Open Access
Peer-reviewed
Research Article
In vitro and in vivo effects of 2,4 diaminoquinazoline inhibitors of the decapping scavenger enzyme DcpS: Context-specific modulation of SMN transcript levels
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Jonathan J. Cherry ,
Contributed equally to this work with: Jonathan J. Cherry, Christine J. DiDonato
Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Project administration, Supervision, Writing – original draft
Current address: RaNA Therapeutics Inc, Cambridge, Massachusetts, United States of America
Affiliation Rare Disease Research Unit, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Christine J. DiDonato ,
Contributed equally to this work with: Jonathan J. Cherry, Christine J. DiDonato
Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Project administration, Resources, Supervision, Writing – original draft, Writing – review & editing
* E-mail: c-didonato@northwestern.edu (CJD); william.gordon@pfizer.com (WG)
Affiliations Department of Pediatrics, Feinberg School of Medicine, Northwestern University, Chicago, Illinois, United States of America, Human Molecular Genetics Program, Ann & Robert Lurie Children’s Hospital, Stanley Manne Research Institute, Chicago, Illinois, United States of America
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Elliot J. Androphy,
Roles Resources, Supervision
Affiliation Department of Dermatology, Indiana University School of Medicine, Indianapolis, Indiana, United States of America
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Alessandro Calo,
Roles Investigation
Affiliation Rare Disease Research Unit, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Kyle Potter,
Roles Data curation, Investigation
Affiliations Department of Pediatrics, Feinberg School of Medicine, Northwestern University, Chicago, Illinois, United States of America, Human Molecular Genetics Program, Ann & Robert Lurie Children’s Hospital, Stanley Manne Research Institute, Chicago, Illinois, United States of America
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Sara K. Custer,
Roles Investigation
Affiliation Department of Dermatology, Indiana University School of Medicine, Indianapolis, Indiana, United States of America
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Sarah Du,
Roles Data curation, Formal analysis, Investigation
Affiliation Precision Medicine, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Timothy L. Foley,
Roles Investigation
Affiliations Pharmaceutical Sciences, Pfizer Worldwide Research and Development, Groton, Connecticut, United States of America, Primary Pharmacology Group, Pfizer Worldwide Research and Development, Groton, Connecticut, United States of America
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Ariamala Gopalsamy,
Roles Conceptualization, Formal analysis, Writing – review & editing
Affiliation Medicine Design, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Emily J. Reedich,
Roles Investigation
Affiliations Department of Pediatrics, Feinberg School of Medicine, Northwestern University, Chicago, Illinois, United States of America, Human Molecular Genetics Program, Ann & Robert Lurie Children’s Hospital, Stanley Manne Research Institute, Chicago, Illinois, United States of America
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Susana M. Gordo,
Roles Investigation
Current address: RaNA Therapeutics Inc, Cambridge, Massachusetts, United States of America
Affiliation Rare Disease Research Unit, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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William Gordon ,
Roles Data curation, Formal analysis, Investigation
* E-mail: c-didonato@northwestern.edu (CJD); william.gordon@pfizer.com (WG)
Affiliation Precision Medicine, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Natalie Hosea,
Roles Conceptualization, Formal analysis
Current address: Takeda California, Global DMPK, San Diego, California, United States of America
Affiliation Pharmacokinetics and Drug Metabolism, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Lyn H. Jones,
Roles Conceptualization, Methodology
Affiliation Medicine Design, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Daniel K. Krizay,
Roles Investigation
Affiliation Rare Disease Research Unit, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Gregory LaRosa,
Roles Conceptualization, Formal analysis, Funding acquisition, Project administration, Supervision
Affiliation Rare Disease Research Unit, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Hongxia Li,
Roles Investigation
Affiliation Department of Dermatology, Indiana University School of Medicine, Indianapolis, Indiana, United States of America
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Sachin Mathur,
Roles Investigation
Affiliation Business Technology, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Carol A. Menard,
Roles Investigation
Affiliations Pharmaceutical Sciences, Pfizer Worldwide Research and Development, Groton, Connecticut, United States of America, Primary Pharmacology Group, Pfizer Worldwide Research and Development, Groton, Connecticut, United States of America
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Paraj Patel,
Roles Investigation
Affiliation Rare Disease Research Unit, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Rebeca Ramos-Zayas,
Roles Investigation, Methodology, Supervision
Affiliation Rare Disease Research Unit, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Anne Rietz,
Roles Investigation
Affiliation Department of Dermatology, Indiana University School of Medicine, Indianapolis, Indiana, United States of America
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Haojing Rong,
Roles Conceptualization, Formal analysis
Affiliation Pharmacokinetics and Drug Metabolism, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Baohong Zhang,
Roles Investigation
Affiliation Clinical Genetics, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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Michael A. Tones
Roles Conceptualization, Data curation, Formal analysis, Funding acquisition, Investigation, Methodology, Project administration, Resources, Supervision, Writing – original draft, Writing – review & editing
Affiliation Rare Disease Research Unit, Pfizer Worldwide Research and Development, Cambridge, Massachusetts, United States of America
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In vitro and in vivo effects of 2,4 diaminoquinazoline inhibitors of the decapping scavenger enzyme DcpS: Context-specific modulation of SMN transcript levels
- Jonathan J. Cherry,
- Christine J. DiDonato,
- Elliot J. Androphy,
- Alessandro Calo,
- Kyle Potter,
- Sara K. Custer,
- Sarah Du,
- Timothy L. Foley,
- Ariamala Gopalsamy,
- Emily J. Reedich
- Published: September 25, 2017
- https://doi.org/10.1371/journal.pone.0185079