Huntington’s disease phenotypes are improved via mTORC1 modulation by small molecule therapy

Sophie St-Cyr; Daniel D. Child; Emilie Giaime; Alicia R. Smith; Christine J. Pascua; Seung Hahm; Eddine Saiah; Beverly L. Davidson

doi:10.1371/journal.pone.0273710

Loading metrics

Open Access

Peer-reviewed

Research Article

Huntington’s disease phenotypes are improved via mTORC1 modulation by small molecule therapy

Sophie St-Cyr,

Roles Conceptualization, Data curation, Formal analysis, Methodology, Supervision, Validation, Writing – original draft, Writing – review & editing

Affiliation The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, PA, United States of America

https://orcid.org/0000-0003-2448-9202

⨯
Daniel D. Child,

Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Supervision, Writing – original draft, Writing – review & editing

Affiliations The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, PA, United States of America, The Perelman School of Medicine, The University of Pennsylvania, Philadelphia, PA, United States of America
⨯
Emilie Giaime,

Roles Data curation, Investigation

Affiliation Navitor Pharmaceuticals Inc., Cambridge, MA, United States of America
⨯
Alicia R. Smith,

Roles Investigation

Affiliation The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, PA, United States of America
⨯
Christine J. Pascua,

Roles Investigation, Methodology

Affiliation Division of Cardiology, The Children’s Hospital of Philadelphia, Philadelphia, PA, United States of America
⨯
Seung Hahm,

Roles Formal analysis

Affiliation Navitor Pharmaceuticals Inc., Cambridge, MA, United States of America
⨯
Eddine Saiah ,

Roles Supervision, Writing – review & editing

* E-mail: davidsonbl@chop.edu (BLD); esaiah@navitorpharma.com (ES)

Affiliation Navitor Pharmaceuticals Inc., Cambridge, MA, United States of America
⨯
Beverly L. Davidson

Roles Conceptualization, Data curation, Formal analysis, Methodology, Project administration, Supervision, Writing – review & editing

* E-mail: davidsonbl@chop.edu (BLD); esaiah@navitorpharma.com (ES)

Affiliations The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, PA, United States of America, The Perelman School of Medicine, The University of Pennsylvania, Philadelphia, PA, United States of America

https://orcid.org/0000-0002-3715-6290

⨯

Huntington’s disease phenotypes are improved via mTORC1 modulation by small molecule therapy

Sophie St-Cyr,
Daniel D. Child,
Emilie Giaime,
Alicia R. Smith,
Christine J. Pascua,
Seung Hahm,
Eddine Saiah,
Beverly L. Davidson

Published: August 29, 2022
https://doi.org/10.1371/journal.pone.0273710

Reader Comments (0)

Post a new comment on this article

Subject Areas
?

For more information about PLOS Subject Areas, click here.
We want your feedback. Do these Subject Areas make sense for this article? Click the target next to the incorrect Subject Area and let us know. Thanks for your help!

Heart
Is the Subject Area "Heart" applicable to this article?

Thanks for your feedback.
Mouse models
Is the Subject Area "Mouse models" applicable to this article?

Thanks for your feedback.
Neostriatum
Is the Subject Area "Neostriatum" applicable to this article?

Thanks for your feedback.
Leucine
Is the Subject Area "Leucine" applicable to this article?

Thanks for your feedback.
Cardiomyocytes
Is the Subject Area "Cardiomyocytes" applicable to this article?

Thanks for your feedback.
Immunoblotting
Is the Subject Area "Immunoblotting" applicable to this article?

Thanks for your feedback.
Huntington disease
Is the Subject Area "Huntington disease" applicable to this article?

Thanks for your feedback.
Fibrosis
Is the Subject Area "Fibrosis" applicable to this article?

Thanks for your feedback.