Single Dose of CRISPR Gene Therapy May Succeed as Long-term Treatment for DMD, Mouse Study Shows
A single dose of gene therapy based on CRISPR-Cas9 may successfully treat Duchenne muscular dystrophy (DMD) in the long term or even permanently, a preclinical study in mice shows. The findings shed light on safety factors that may affect the long-term efficacy of this gene therapy, and provide new evidence…