Rescue Of Dystrophic Muscle Through U7 Sn Rna–Mediated Exon Skipping

727 words - 3 pages

Duchenne muscular dystrophy (DMD) is a form of muscular dystrophy and is recessive and X-linked. This means that females can be carrier of the disease while males are affected by this disease. No female would be affected by this disease, because affected males would not be able to reproduce. DMD is a form of muscular dystrophy that is caused by a mutation in the dystrophin gene, which encodes for dystrophin, a cytoskeletal protein that forms a protein complex and connects the cytoskeleton of muscle fibers to the extracellular matrix. This protein is needed to maintain the membrane stability of the muscle fibers and without this stability patients would have weakened muscles and a loss of ...view middle of the document...

They chose to use a 24-nucleotide long antisense sequence (BP22) for the splicing branch point in intron 22 and a 20-nucleotide long antisense sequence (SD23) that matches with the U1 binding region in intron 23. BP22 splices at the 3’ end of the pre-mRNA and SD23 splices at the 5’ end of the pre-mRNA of dystrophin.
This created a modified U7 gene, which they introduced into a Adeno-associated virus vector (AAV) along with the natural promotor and 3’ components. These vectors are used to infect muscle cells, where the antisense sequences of the modified gene can be transcribed, thus triggering exon-skipping of the dystrophin pre-mRNA and this eventually leads to an increase of the shorther dystrophin protein.

The procedures used by the authors resulted in production of rescued dystrophin.
2 weeks after injection we see mRNA of dystrophin without exon 23 and this continues for at least 11 weeks, but within those 2 weeks there isn’t much rescued dystrophin yet. With the Western blot of the protein extracted from the muscles of the mdx mouse we clearly see that the process of production of rescued dystrophin starts approximately 2 weeks...

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