Improved Dystrophin Expression and Cardiac Function in Mdx Pups Using CRISPR-Cas

Prior studies have shown the use of CRISPR-Cas gene editing as a alternative treatment for Duchenne Muscular Dystrophy (DMD) that can normalise dystrophin expression, and skeletal and cardiac function in diseased mouse models (mdx pups).


However, there are questions regarding the efficacy of the treatment option, and whether the gene edited DNA is a viable long term treatment and is safe to use.


Now an article has been published in Molecular Therapy, whereby Scientists injected an AAVrh.74 vector containing Cas9 with the specific gRNA to target intron 20 and 23 in the mdx pups, and then 19 months later the pups were re-examined.


It was found that the gene editing in the mdx pups had increased dystrophin expression levels which is reduced in Duchenne Muscular Dystrophy, as well as improving the cardiac function, with no serious complications as a result of the gene editing, it must be noted that these genetic changes were life long changes to the genome.


The Scientists of this article, declared that these results provide evidence that in vivo gene editing is a safe therapy option for DMD and many more diseases.


References:

https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(19)30215-1


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