Gene therapy has shown considerable promise as a treatment for Friedreich’s ataxia, but current approaches face important challenges. Delivering therapies to affected tissues, particularly the central nervous system, can be difficult, and uncontrolled production of frataxin may raise safety concerns. These limitations highlight the need for approaches that can restore frataxin while maintaining appropriate levels of gene expression.
This project will develop a next-generation gene therapy that combines advanced viral delivery technologies with synthetic genetic circuits designed to regulate frataxin expression. These built-in mechanisms aim to ensure that cells produce the right amount of frataxin, reducing the risk of overexpression while improving delivery to affected tissues.
By incorporating programmable regulation directly into the therapy, this research aims to broaden the therapeutic window and enhance performance across multiple organ systems. If successful, the work will support development of a candidate therapy designed to provide widespread, well-regulated frataxin replacement throughout the body.