Gene editing on dogs shows promise in treating muscular dystrophy

Scientists used the gene editing technology CRISPR in dogs to treat an incurable genetic disorder. And this could hold the key for treating Duchenne muscular dystrophy in humans.

For the uninitiated, muscular dystrophy is a group of genetic disease that causes progressive weakness and loss of muscle mass. And a more severe form of it known as Duchenne muscular dystrophy (DMD) where the symptoms of muscle weakness start as early as in four-year olds and the condition worsens rather quickly.

But good news is coming from University of Texas Southwestern Medical Center as a group of researchers repaired DMD mutation in dogs to restore some muscle functionality in them through gene-editing technology CRSIPR.

There have been several successful attempts of using CRISPR on mice with positive results, but this new group of scientists wanted to test if the same can be done on larger animals.

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For the study, the researchers bred four beagles with DMD symptoms, and then used CRISPR to edit exon 51, the dystrophin gene that codes for the protein, making it error free. They believed that those errors would cause the body's affected cells to skip exon 51 altogether. And expected that though reduced, but there would be functional level of dystrophin production in dogs' bodies.

The dogs were then given CRISPR via a harmless virus when they were one month old. After six to eight weeks, the researchers found that dystrophin levels were at 92 per cent and 56 per cent normal levels in the heart and diaphragm respectively.

Lead author Leonela Amoasii said that their strategy was different from other therapeutic approaches for DMD as they edit the mutation that leads to the disease and restores normal expression of the repaired dystrophin. However, she added that they need to more tests before using it clinically.

There are only 15 per cent of people with DMD have the particular mutation that was corrected in the dogs, and there are other gene edits needed to help out about 3,00,000 boys worldwide believed to be suffering from the disease.

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