Frataxin expression constructs having engineered promoters and methods of use thereof

Abstract

This invention relates to compositions and methods for modifying, for example enhancing, the performance of ataxia (FXN) in vitro and / or in vivo, including (but not limited to) employing engineered promoters. Such compositions include delivery via administration of adeno-associated virus (AAV) particles. These compositions and methods of the invention are suitable for treating individuals diagnosed with or suspected of having Friedreich's ataxia, or another neuromuscular or neurological condition caused by or associated with a deficiency in the amount and / or function of ataxia or a reduction in the performance or protein content of ataxia.