Possible breakthrough for Muscular Dystrophy?

A gene-editing tool called “CRISPR” has been used for the first time to help dogs with Duchenne Muscular Dystrophy. The hope is that this tool will be effective in people with Muscular Dystrophy as well.

Muscular Dystrophy is a genetic disease that affects over 20,000 patients, mostly boys, who lose their ability to walk usually by their 20s.  The disease also affects dogs.

Scientists have tried many medicines in the past to help these patients, but have had limited success.

CRISPR technology may be a possible breakthough for this disease.  CRISPRs are a type of DNA found in bacteria, and they can edit very precise locations of DNA in cells.    The enzymes that are produced by CRISPR can be programed to bind to the patient’s DNA and shut off the gene that is causing a problem.

The researchers in this study used the CRISPR gene-editing system to restore the production of dystrophin in four dogs, the protein needed for healthy muscle function.

The next step is to treat more dogs with Muscular Dystrophy and continue to study this new treatment for a year, with the hopes of then trying the treatment in humans.

(CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats.)



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