CRISPR Enhancement Cures Genetic Disease in Mice

Scientists in Switzerland have used an enhanced CRISPR-Cas gene editing technology to correct the gene mutation that causes the metabolic disorder phenylketonuria (PKU), in mice, and restore blood levels of the amino acid phenylalanine, to normal. Reporting on the technology in Nature Medicine , the researchers say their results offer “compelling evidence” that the method can be used to reverse the disease. This approach has great potential for application in humans, claims study hea