Translation-Guided RNA engineering Platform for Cardiac disease

Obiettivo

Many inherited diseases arise not merely because a gene is mutated, but because cells no longer produce enough of the right protein. Because pathogenic variants are often highly diverse and spread across the gene, correcting each mutation individually is difficult to scale. TRANSLATE introduces a new therapeutic logic: precise translation engineering at the RNA level to reinstate physiological protein output without genomic modification in genetic dosage disorders. TRANSLATE builds on discoveries from the ERC onsolidator Grant TRANSCEND, which studies how cells control the translation of selected mRNAs into proteins. Our central idea is that RNA contains regulatory information that determines stability, localization, translation efficiency, and protein output. If this regulatory logic can be read, modeled, and rewritten, endogenous RNAs may be redirected to restore protein output without altering the genome. TRANSLATE will combine systems RNA biology, quantitative modeling, and machine learning to understand the RNA regulatory logic that determines mRNA stability, translation, and protein output. The project aims to create a path toward mutation-agnostic RNA therapeutics for genetic dosage disorders.