New gene editing method shows promise for muscular dystrophy

15 August 2014

Researchers at the University of Texas Southwestern Medical Center have successfully used a new gene editing method to correct the mutation that leads to Duchenne muscular dystrophy (DMD).

From a mouse model of the condition, researchers used a technique called CRISPR/Cas9-mediated genome editing. This can remove a single mutation in DNA, which then allows it to repair mechanisms to replace it with a healthy version of the gene.

Dr Eric Olson, director of the Hamon Center for Regenerative Science and Medicine at Southwestern and chairman of Molecular Biology, said the benefit of this is that it can permanently correct the "defect" in a gene.

Using this method, the team were able to correct the genetic defect in the mouse model of DMD and prevent the development of features of the disease in boys.

Dr Olson said this is "very important for possible clinical application of this approach in the future".

Although scientists have known the gene behind DMD for nearly 30 years, there are no treatments that can cure the condition, which destroys muscle fibres and replaces them with fatty tissue.

Posted by Philip Briggs​


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