NeurosciencebioRxivPreprint
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…·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.