The first gene-silencing drug wins FDA approval

A Nobel Prize–winning discovery — that small double-stranded RNA molecules can silence genes by interrupting the translation of DNA’s instructions into proteins — is finally delivering on its medical promise. The first drug that takes advantage of this natural biological process, called RNA interference, was approved August 10 by the U.S. Food and Drug Administration. […]

CRISPR gene editing relieves muscular dystrophy symptoms in dogs

Gene editing can reverse muscular dystrophy in dogs. Using CRISPR/Cas9 in beagle puppies, scientists have fixed a genetic mutation that causes muscle weakness and degeneration, researchers report online August 30 in Science. Corrections to the gene responsible for muscular dystrophy have been made before in mice and human muscle cells in dishes, but never in […]