Core Insights - The article discusses a significant breakthrough in the treatment of Rett Syndrome, a rare genetic disorder caused by mutations in the MeCP2 gene, primarily affecting girls at a rate of 1 in 10,000 [6] - Researchers at Harvard's Wyss Institute have identified Vorinostat, an FDA-approved drug for skin T-cell lymphoma, as a promising therapeutic option for Rett Syndrome, demonstrating disease-repair capabilities in preclinical models [2][8] Rett Syndrome Challenges - Rett Syndrome is characterized by complex cognitive and physical impairments due to a single gene mutation, leading to multiple organ dysfunctions, including neurological, digestive, muscular, and immune system issues [6] - The complexity of the disease, involving changes in multiple gene expressions, complicates the development of effective treatments based on single drug targets [6] Research Methodology - The research team utilized an AI-driven system called nemoCAD to revolutionize traditional drug development approaches by: 1. Constructing animal models using African clawed frog tadpoles to simulate human MeCP2 gene mutations, replicating various symptoms of Rett Syndrome [7] 2. Conducting whole-genome analysis to compare gene expression changes between healthy and affected tadpoles [7] 3. Performing drug reverse screening based on gene expression profiles from the NIH LINCS database to identify candidate drugs that could reverse pathological changes [7] Drug Discovery and Results - Vorinostat, a histone deacetylase inhibitor, showed the most promising results, significantly reversing abnormalities in neurological, intestinal, and muscular systems in affected tadpoles [8] - The research indicates that Vorinostat could potentially be the first curative therapy for Rett Syndrome, leveraging a unique AI-based drug discovery approach combined with innovative disease modeling [11] Clinical Trials and Future Plans - Unravel Biosciences, a biotech company incubated by Wyss Institute, plans to initiate clinical trials in Colombia in 2025, involving 15 female patients [12] - The trials will adopt an n-of-1 personalized experimental design, adjusting treatment plans for each patient, and will advance the proprietary formulation RVL-001 of Vorinostat [12]