...dystrophy (DMD), an X-linked recessive disorder affecting approximately 1 in 5000 males. DMD is caused ...difference in DMD, and it’s estimated that exon skipping therapies would be applicable to 80% of DMD patients...feasible case of DMD. If the safety and efficacy of this approach can be optimized, DMD could become one...age. Despite the amount of research conducted on DMD, there is still no good treatment. The dystrophin...be functional, exon skipping is a good option for DMD treatment. Clinical trials have used oligonucleotide...paired zinc finger nucleases to remove exon 51 in DMD patient myoblasts. They observed a 13% removal rate...exons 45-55 in patient myoblasts; when injected into DMD mice, these cells expressed functional dystrophin...

...SON-mEGFP AICSDP-82 mEGFP SON Nuclear speckles 159744 DMD-mEGFP AICSDP-55 mEGFP Dystrophin Costameres 159745...Institute ID Tag Protein Structure 87420 PXN-EGFP AICSDP-1 EGFP Paxillin Matrix Adhesions 87421 TUBA1B-mEGFP ...TJP1-mEGFP AICSDP-23 mEGFP Tight junction protein ZO-1 Tight junctions 91565 AAVS1-mEGFP AICSDP-35 mEGFP ...Centrioles 101782 LAMP1-mEGFP AICSDP-19 mEGFP LAMP-1 Lysosome 101783 MAP1LC3B-mEGFP AICSDP-25 mEGFP Autophagy-related...101786 ST6GAL1-mEGFP AICSDP-26 mEGFP Sialyltransferase 1 Golgi 109122 NPM1-mEGFP AICSDP-50 mEGFP Nucleophosmin... HIST1H2BJ-mEGFP AICSDP-52 mEGFP Histone H2B type 1-J Histones 109119 CTNNB1-mEGFP AICSDP-47 mEGFP Beta-catenin...