Jan 04, 2016 11:46 PM EST
Gene therapy could help create muscular dystrophy therapy
A new study has revealed that the gene editing technique, Crispr-Cas9, could help in developing a treatment for muscular dystrophy, dddmag reports.
Muscular dystrophy is caused by a genetic mutation that inhibits the growth of proteins called dystrophin, which help in the production of healthy muscle mass.
The study was published in the journal Science.
Three independent research groups from Duke University, Harvard University, and the University of Texas Southwestern Medical Center conducted the experiment on a group of mice.
According to New York Times, the dystrophin protein anchors each muscle fiber to the membrane that encloses the muscle-fiber bundle. The dystrophin gene spreads across about 1 percent of the X chromosome and is the largest in the human genome.
The teams were led by Charles A. Gersbach of Duke University, Eric N. Olson of the University of Texas Southwestern Medical Center and Amy J. Wagers of Harvard University.
"The papers are pretty significant," said Louis M. Kunkel, a muscular dystrophy expert at Boston Children's Hospital who discovered the dystrophin gene in 1986.
Crispr-Cas9 inserts healthy genes in the place of defective ones to correct genetic disorders. With the Crispr-Cas9, the researchers were able to remove an exon, which is a damaged stretch of DNA. This helped the mice gain strength since the muscle cells produced a functional, but shortened, dystrophin strand.
Even though the three teams filed patents for their discoveries, more research needs to be conducted before clinical trials can commence.
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