Could CRISPR/Cas9 Therapy Work for Duchenne Muscular Dystrophy?


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.

Muscular Dystrophy News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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  1. jason says:

    Hi, my son has Duchene muscular dystrophy and have been told by his specialist. that crisp/cas9 would benefit him, just wounding when and if this treatment would come to Australia. I’ve been following on emails to help save my son.

    • Dianne Roncal, DMD says:

      Hi Jason, as of now, we do not know any particulars of when this drug will be available outside of the US

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