The “Centro Dino Ferrari” of the University of Milan – Policlinico Hospital has launched a research project aimed at studying the role of gold nanoparticles in stabilizing frataxin (FXN) mRNA, titled “Gold nanoparticles for gene protection in the therapy of Friedreich’s Ataxia” (PROTHEUS). The project aims to decipher the molecular mechanisms by which gold nanoclusters influence FXN mRNA stability. The study will be conducted on neural cell models derived from iPSCs of patients affected by Friedreich’s Ataxia (FRDA).
This study is of particular relevance because it addresses a knowledge gap regarding the post-transcriptional mechanisms of FXN in the central nervous system and validates, for the first time, a specific cellular model for this pathology, paving the way for innovative nanotherapies. The initiative is part of a broader multidisciplinary research network, involving experts in neurology, nanotechnologies, molecular biology, and epitranscriptomics.
Kinetic validation of FXN mRNA half-life using pulse-chase assays.
Mechanistic analysis of RNA-gold nanoparticle interactions and RNA-binding protein recruitment via RIP-qPCR.
Targeted transcriptomic profiling for genomic mRNA stability.
Quantification of oxidative damage to FXN mRNA and correlation with gold nanocluster protection.
To provide validated protocols, datasets on RNA stabilization mechanisms, and the identification of key processes for translational clinical applications in neurodegenerative diseases.
Budget € 350,000
Raised € 69,000
Prof. Yvan Torrente yvan.torrente@unimi.it