DALLAS August 14, 2014 UT Southwestern Medical Center researchers successfully used a new gene editing method to correct the mutation that leads to Duchenne muscular dystrophy (DMD) in a mouse model of the condition.
Researchers used a technique called CRISPR/Cas9-mediated genome editing, which can precisely remove a mutation in DNA, allowing the body's DNA repair mechanisms to replace it with a normal copy of the gene. The benefit of this over other gene therapy techniques is that it can permanently correct the "defect" in a gene rather than just transiently adding a "functional" one, said Dr. Eric Olson, Director of the Hamon Center for Regenerative Science and Medicine at UT Southwestern and Chairman of Molecular Biology.
Using CRISPR/Cas9, the Hamon Center team was able to correct the genetic defect in the mouse model of DMD and prevent the development of features of the disease in boys, which causes progressive muscle weakness and degeneration, often along with breathing and heart complications.
"Our findings show that CRISPR/Cas9 can correct the genetic mutation that leads to DMD, at least in mice," said Dr. Eric Olson, holder of the Pogue Distinguished Chair in Research on Cardiac Birth Defects, the Robert A. Welch Distinguished Chair in Science, and the Annie and Willie Nelson Professorship in Stem Cell Research. "Even in mice with only a subset of corrected cells, we saw widespread and progressive improvement of the condition over time, likely reflecting an advantage of the corrected cells and their contribution to regenerating muscle."
He also pointed out "this is very important for possible clinical application of this approach in the future. Skeletal muscle is the largest tissue in the human body and current gene therapy methods are only able to affect a portion of the muscle. If the corrected tissue can replace the diseased muscle, patients
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UT Southwestern Medical Center