1st DMD Mouse Model to Mimic Heart Disease Confirms Gene Therapy Efficacy

1st DMD Mouse Model to Mimic Heart Disease Confirms Gene Therapy Efficacy

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1st DMD Mouse Model to Mimic Heart Disease Confirms Gene Therapy Efficacy

For the first time, a new mouse model has been shown to mimic the heart disease features — specifically, to progress into late-stage heart failure, similar to humans — of people with Duchenne muscular dystrophy (DMD), a study reports. Moreover, using the new mouse model, researchers gathered promising data supporting the effectiveness of micro-dystrophin gene therapy, currently being tested in clinical trials. The gene therapy was found to rescue heart function and prevent heart failure…

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