AI-enabled drug prediction and gene network analysis reveal therapeutic use of vorinostat for Rett Syndrome in preclinical models.

Journal: Communications medicine
Published Date: Jul 1, 2025

Abstract

BACKGROUND: Many neurodevelopmental genetic disorders, such as Rett syndrome, are caused by a single gene mutation but trigger changes in expression of numerous genes. This impairs functions of multiple organs beyond the central nervous system (CNS), making it difficult to develop broadly effective treatments based on a single drug target. This is further complicated by the lack of sufficiently broad and biologically relevant drug screens, and the inherent complexity in identifying clinically relevant targets responsible for diverse phenotypes that involve multiple organs.

Authors

  • Richard Novak
    Wyss Institute for Biologically Inspired Engineering at Harvard University, Boston, MA, USA.
  • Tiffany Lin
    Smith School of Chemical and Biomolecular Engineering, Cornell University, Ithaca, NY 14853, USA.
  • Shruti Kaushal
    Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.
  • Megan Sperry
    Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.
  • Frederic Vigneault
    Wyss Institute for Biologically Inspired Engineering , Boston, Massachusetts 02115, United States.
  • Erica Gardner
    Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.
  • Sahil Loomba
    Department of Mathematics, Imperial College London.
  • Kostyantyn Shcherbina
    Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.
  • Vishal Keshari
    Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.
  • Alexandre Dinis
    Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.
  • Anish Vasan
    Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.
  • Vasanth Chandrasekhar
    Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.
  • Takako Takeda
    Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.
  • Rahul Nihalani
    Unravel Biosciences, Inc., Boston, MA, USA.
  • Sevgi Umur
    Unravel Biosciences, Inc., Boston, MA, USA.
  • Jerrold R Turner
    Department of Pathology, Brigham and Women's Hospital, Boston, Mass; Division of Gastroenterology, Hepatology, and Endoscopy, Brigham and Women's Hospital, Boston, Mass.
  • Michael Levin
    Department of Biology, Allen Discovery Center at Tufts University, Tufts University, 200 Boston Ave. Suite 4604, Medford, MA, 02155, USA.
  • Donald E Ingber
    1 Wyss Institute for Biologically Inspired Engineering, Harvard University , Boston, Massachusetts.

Keywords

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