FARA NZ and Australia FARA are co-funding the Deverman research project. This research provides hope to NZ families and those throughout the world living with Friedreich Ataxia. We are grateful for the fundraising efforts of the FARA NZ community used to support this important research.
Evaluating novel capsids engineered for efficient CNS transduction, as frataxin gene delivery vehicles
Friedreich ataxia patients experience neurological problems including loss of coordination, speech difficulties, vision and hearing impairments, as well as muscular/skeletal and cardiac abnormalities and often diabetes. These symptoms stem from a gene mutation that results in insufficient production of the protein frataxin. Encouragingly, in mouse models of the disease, restoration of frataxin expression with gene therapy can prevent and even reverse the cardiac abnormalities. While gene therapy is a promising approach to treat the late stage cardiac symptoms of the disease, using this approach to treat the neurological symptoms is more challenging in part because there are currently no gene transfer vehicles that can efficiently deliver the frataxin gene throughout the adult nervous system. To address this need, we have recently developed inactivated, non-replicating, viral vectors that can, after a single intravenous injection, deliver genes throughout the entire mouse nervous system. Importantly, our vector also transfers genes to other sites of frataxin deficiency including skeletal and cardiac muscle and the pancreas. Together these characteristics put our vector in the unique position to boost frataxin expression in the nervous system and other key sites. We aim to test whether frataxin delivery with our novel vectors can prevent the neurological disease progression observed in a mouse model of Friedreich ataxia. If successful, our study would set the stage for more extensive testing in additional preclinical models of Friedreich ataxia. Would you like to know more?Voyager Therapeutics Licenses Novel Gene Therapy CapsidsDr. Ben Deverman on gene therapy