Can a mutated gene for dystrophin not have DMD?
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95 Citations | If this is the case, it may be possible to modify the regulation of utrophin expression as an alternative route to dystrophin gene therapy for sufferers of DMD and/or BMD. |
16 Citations | Therefore, the dystrophin muscle promoter/enhancer sequence represents an alternative for use in gene therapy vectors for the treatment of DMD. |
| Mutations in the DMD gene are thought to be responsible, with the shorter isoforms of dystrophin implicated in its molecular brain pathogenesis. | |
| The observation of hybrid repeat reconstitution in some cases provides insight into phenotype-genotype correlations in dystrophin diseases and possible strategies for gene therapy. | |
15 Citations | The distribution of deletions in dystrophin gene is associated with the phenotype of DMD/BMD. |
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