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Comparison of major approaches for genetic correction for DMD. Four different antisense oligonucleotides (AON) have been approved for use in the US. These target specific DMD mutations. Adeno-associated viral gene therapy is being investigated in ongoing clinical trials and aims to produce micro-dystrophin in skeletal and cardiac muscle. Gene editing is also contemplated for DMD; gene editing has been tested in preclinical models, including a large animal model for DMD.
