Target-agnostic discovery of Rett Syndrome therapeutics by coupling computational network analysis and CRISPR-enabled in vivo disease modeling
R. Novak, Tiffany Lin, S. Kaushal, Megan M. Sperry, Frédéric Vigneault, E. Gardner, Sahil Loomba, K. Shcherbina, Vishal Keshari, A. Dinis, Anish Vasan, V. Chandrasekhar, Takako Takeda, Jerrold R. Turner, Michael G. Levin, Donald E. Ingber
This paper identifies vorinostat as a potential repurposed therapy for Rett syndrome by combining computational network analysis with CRISPR-edited Xenopus models, showing efficacy across multiple organ systems.
bioRxiv · Mar 20, 2022 · PreprintNeuroscience