RNA-based translation activators for targeted gene upregulation. A molecular technology called "translation-activating RNAs" (taRNAs) shows promise in treating diseases caused by haploinsufficiency, where one functional gene copy is absent. taRNAs are small molecules designed to bind to specific mRNA molecules, regulating their translation into proteins. This approach was successful in increasing mRNA levels for PTEN and ABCA7, genes associated with tumor suppression and Alzheimer's disease. In a human sample of triple-negative breast cancer, taRNAs slowed tumor growth. The technology also restored protein levels in SYNGAP1, a gene linked to epilepsy and impaired neurodevelopment. Read more: https://lnkd.in/dNqvbgcc #rna #tumor #biology #alzheimer #medicalresearch
CEO Founder at Within[u]: Consciousness Ai | Digital Healthcare
11moRNA-based translation activators is a game-changer in bioinformatics and genetic medicine. By specifically targeting and upregulating gene expression, as seen with key genes like PTEN and ABCA7, taRNAs show great potential in treating complex diseases, including cancer and neurodevelopmental disorders. This could be the future of targeted genetic therapies.