COMPOSITIONS AND METHODS OF USE THEREOF FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
The present disclosure provides, in part, a method of treating subjects with Duchenne muscular dystrophy (DMD) via gene editing approaches that induce exon deletion(s) to restore the reading frame of the dystrophin gene, thereby restoring dystrophin protein activity. The invention also provides comp...
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| Main Author | |
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| Format | Patent |
| Language | English |
| Published |
11.04.2024
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| Subjects | |
| Online Access | Get full text |
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| Abstract | The present disclosure provides, in part, a method of treating subjects with Duchenne muscular dystrophy (DMD) via gene editing approaches that induce exon deletion(s) to restore the reading frame of the dystrophin gene, thereby restoring dystrophin protein activity. The invention also provides compositions comprising adeno-associated viral vectors, an RNA-guided nucleases, nickases or DNA endonucleases, and guide RNAs for use in the treatment of subjects with DMD, or subjects with other neuromuscular genetic diseases or disorders. |
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| AbstractList | The present disclosure provides, in part, a method of treating subjects with Duchenne muscular dystrophy (DMD) via gene editing approaches that induce exon deletion(s) to restore the reading frame of the dystrophin gene, thereby restoring dystrophin protein activity. The invention also provides compositions comprising adeno-associated viral vectors, an RNA-guided nucleases, nickases or DNA endonucleases, and guide RNAs for use in the treatment of subjects with DMD, or subjects with other neuromuscular genetic diseases or disorders. |
| Author | Jaskula-Ranga, Vinod |
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| Snippet | The present disclosure provides, in part, a method of treating subjects with Duchenne muscular dystrophy (DMD) via gene editing approaches that induce exon... |
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| Title | COMPOSITIONS AND METHODS OF USE THEREOF FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY |
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