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A promissing mouse model for Friedreich Ataxia progressing like human patients

Many groups worked on the creation of FRDA mouse models by decreasing the mouse frataxin or knocking it out, or by introducing a transgene with a human frataxin with long GAA repeat. Most of the mouse models are limited to one problem, either neurologic or cardiac symptoms, and, for those that have both, generally these symptoms are too severe, and mice have a very short life span, which does not reflect the human disease's progression. Jackson Laboratories Inc. developed a new mouse model that has 800 GAA repeats. The authors report here that the YG8-800 mice show progressively worsening locomotion and balance phenotypes starting at 11 to 20 weeks depending on the behavioral test. Moreover, these mice showed at 26 weeks a heart hypertrophy characterized by an increased heart to body weight ratio. YG8-800 mice are thus currently a promising mouse model for FRDA.

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