February 2018
Vol 4, Issue 2

About The Cover

Cover image expansion

ONLINE COVER Researchers working in heart muscle cells derived from human stem cells have used CRISPR/Cas9 to correct mutations in clustered sections of the X-linked dystrophin gene. Through this technique, they were able to improve both muscle and cardiac abnormalities associated with Duchenne muscular dystrophy (DMD), a genetic disorder that is caused by various kinds of genetic mutations and results in the degeneration of muscle cells. According to the authors, their approach could salvage muscle function in up to 60% of DMD patients. Studies of the DMD gene to date have pinpointed clustered mutations responsible for DMD abnormalities in “hot spot” areas. Chengzu Long et al. proposed a method that takes advantage of this fact, allowing for the removal of a wide range of mutations in these regions, an approach they call "myoediting." Using CRISPR/Cas9 with single-guide RNAs, the researchers performed myoediting in heart muscle cells derived from induced pluripotent stem cells and efficiently restored expression of the dystrophin protein in these cardiac muscle cells. CREDIT: SPL/SCIENCE SOURCE