A genetic muscle disorder in a living adult mammal has, for the first time ever, been treated by scientists by using a CRISPR gene-editing tool. The development by the scientists is being praised worldwide and is being seen as a promising medical breakthrough that could soon lead to the formation of human therapies.
For the study, three independent research groups from Duke University, Harvard University, and the University of Texas Southwestern Medical Center conducted experiments on a group of mice.
Published in the journal Science, a new study says the gene-editing technique, Crispr-Cas9, could contribute considerably in developing a treatment for muscular dystrophy, which is caused by a genetic mutation that inhibits the growth of proteins called dystrophin. These proteins, in turn, help in the production of healthy muscle mass.
A researcher from the Duke University, Charles A. Gersbach, said, “There is still a significant amount of work to do to translate this to a human therapy and demonstrate safety”.
Gersbach said these results coming from their first experiments were very exciting. From here, they would be optimizing the delivery system, evaluating the approach in more severe models of DMD, and assessing efficiency and safety in larger animals with the eventual goal of getting into clinical trials, he added.