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

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 ef...

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Main Authors: Richard Novak, Tiffany Lin, Shruti Kaushal, Megan Sperry, Frederic Vigneault, Erica Gardner, Sahil Loomba, Kostyantyn Shcherbina, Vishal Keshari, Alexandre Dinis, Anish Vasan, Vasanth Chandrasekhar, Takako Takeda, Rahul Nihalani, Sevgi Umur, Jerrold R. Turner, Michael Levin, Donald E. Ingber
Format: Article
Language:English
Published: Nature Portfolio 2025-07-01
Series:Communications Medicine
Online Access:https://doi.org/10.1038/s43856-025-00975-8
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