New gene editing method may correct muscular dystrophy

A new study has used a new gene editing method to correct the mutation that leads to Duchenne muscular dystrophy (DMD) in a mouse model.

The researchers from UT Southwestern Medical Center used a technique called CRISPR/Cas9-mediated genome editing, which could precisely remove a mutation in DNA, allowing the body's DNA repair mechanisms to replace it with a normal copy of the gene.

Eric Olson, Chairman of Molecular Biology, said that the benefit of this over other gene therapy techniques was that it could permanently correct the "defect" in a gene rather than just transiently adding a "functional" one.

He asserted that out that this was very important for possible clinical application of this approach in the future, as skeletal muscle is the largest tissue in the human body and current gene therapy methods were only able to affect a portion of the muscle.

Olson added that if the corrected tissue could replace the diseased muscle, patients might get greater clinical benefit.

The research is published online in the journal Science.