60. Intramuscular and Systemic Induction of the N-Truncated Dystrophin by Out-Of-Frame Exon 2 Skipping Restores Muscle Function in the Dup2 Mouse, Providing Further Support for a Therapeutic Pathway for 5’ DMD Mutations

Molecular Therapy - United States
doi 10.1016/s1525-0016(16)33665-6
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Elsevier BV

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