Gene-Editing Breakthrough Saves an Infant with a Rare Disease

Gene-Editing Breakthrough Saves an Infant with a Rare Disease

CRISPR therapies are moving from proof-of-concept to real-world treatments for a growing list of diseases.

Small molecule makes RDEB gene editing more accurate: Study

Researchers used a small molecule called M3814 for more accurate editing of genetic mutations causing recessive dystrophic epidermolysis bullosa (RDEB), according to a new study. This method enabled skin cells from three patients to restore their production of type VII collagen (C7) protein, which they lacked, the researchers noted. M3814 works by inhibiting DNA-dependent protein kinase, an enzyme that repairs breaks in DNA. Using M3814, the res…

A baby with a rare and incurable disease became the world's first patient to have its edited DNA, which allowed its recovery to date.