AAV-Mediated CRISPR/Cas9 Gene Editing for Correction of DMD Exon Duplications in Patients with Muscular Dystrophy

Gene therapy experts at Nationwide Children’s have developed an AAV-mediated CRISPR/Cas9 gene editing method for the correction of exon duplications in patients with DMD (Duchenne muscular dystrophy). This therapy has the potential to permanently corrects DMD by stopping and potentially reversing the progression of muscle wasting and fibrosis in affected individuals. Currently about 11% of DMD cases are caused by exon duplications and our experts plan to use this invention to correct for this underlying cause within muscle tissues.

Patents

Patent # Title Country
18/279,169 Products and Methods for Treatment of Dystrophin-Based Myopathies Using CRISPR/CAS9 to correct DMD Exon Duplications United States of America
3212108 Products and Methods for Treatment of Dystrophin-Based Myopathies Using CRISPR/CAS9 to correct DMD Exon Duplications Canada
2022229489 Products and Methods for Treatment of Dystrophin-Based Myopathies Using CRISPR/CAS9 to correct DMD Exon Duplications Australia

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