Advances in Gene and Cellular Therapy in Friedreich Ataxia.

Abstract excerpt

Friedreich ataxia is a rare, autosomal recessive neurogenerative disorder caused by mutations to the frataxin (FXN) gene resulting in loss of functional FXN protein. Applications of cutting-edge genetic and cellular therapies expand therapeutic options for patients with rare, genetic diseases including Friedreich ataxia. Multiple investigational techniques and strategies seek to permanently alter the disease...

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