FRDA

Friedreich’s Ataxia (FRDA) Research

Overview

Friedreich’s Ataxia (FRDA) is a rare, inherited neurodegenerative disorder caused by insufficient expression of the frataxin (FXN) gene. This results in progressive damage to the nervous system and heart, affecting mobility, coordination, and overall quality of life. The Yokota Lab is dedicated to developing innovative therapies that target the root cause of FRDA and improve patient outcomes.

Our Therapeutic Approach

Our research aims to upregulate FXN expression using antisense oligonucleotides (ASOs) designed to target the GAA repeat expansion in the FXN gene intron. These expansions silence FXN expression, and our ASOs work by modifying transcriptional interference at the locus.

We also use lipid nanoparticles (LNPs) to deliver ASOs efficiently into target tissues, including cardiac and neuronal cells — two of the most affected systems in FRDA.

Our Goal

Our ultimate goal is to develop a safe and effective RNA-targeted therapy for FRDA, with potential to move toward clinical translation in partnership with patients, families, and advocacy groups.

Last updated: 23 April, 2025