FXN Gene Therapy Treats Heart Problems in New FA Mouse Model
A gene therapy that delivers a healthy version of the FXN gene can correct heart problems in a new mouse model with cardiac-specific symptoms of Friedreich’s ataxia[2] (FA), a study reports.[1] This animal model more accurately mirrors the early cardiac abnormalities seen in FA patients, the researchers wrote, providing new opportunities to study FA symptoms and investigate disease-modifying therapies. The...
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