Could CRISPR/Cas9 Therapy Work for Duchenne Muscular Dystrophy?

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by Wendy Henderson |

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A powerful new genome editing tool clustered regularly interspaced short palindromic repeats or CRISPR/Cas9 could offer a solution in the treatment of Duchenne muscular dystrophy (DMD). CRISPR/Cas9 mimics the way bacteria works by eliminating the DNA of invading viruses and replacing it with part of its own DNA, slicing into the DNA and rebuilding it from within. Find out more about CRISPR/Cas9 here.

MORE: Stem cell therapy shows promise in treating DMD-linked heart disease. 

Research is currently underway in three separate laboratories for treatment of DMD, where CRISPR/Cas9 is being used to investigate and fix mutated dystrophin genes in mouse models.

The mice treated with CRISPR/Cas9 showed greater grip strength, a reversal of muscle necrosis, reduced levels of creatine kinase in the blood, and decreased fibrosis.

MORE: CRISPR-Cpf1 correct Duchenne mutated and restores dystrophin expression, a study finds.

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