The breakthrough genome editing tool known as CRISPR (clustered regularly interspaced short palindromic repeats) has been used by three separate research groups to treat postnatal Duchenne Muscular Dystrophy (DMD) mice. To correct DMD by skipping mutant dystrophin exons in postnatal muscle tissue in vivo, the investigators used adeno-associated virus-9 (AAV9) to deliver the CRISPR/Cas9 system to mdx mice in order to remove the mutated exon 23 from the dystrophin gene. Following local or systemic delivery, this excision resulted in expression of the modified dystrophin gene, partial recovery of functional dystrophin protein in skeletal myofibers and cardiac muscle, improvement of muscle biochemistry, and significant enhancement of muscle force. Furthermore, this treatment generated a pool of endogenously corrected myogenic precursors in mdx mouse muscle. CRISPR/Cas9-based genome editing provides a potential means of correcting mutations responsible for DMD and other postnatal monogenic disorders.
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