Therapeutic Potential of CRISPR Research Raising Safety and Ethics Questions

Abstract

CRISPR/Cas9-based genome editing is a potential treatment for Duchenne muscular dystrophy (DMD), according to Charles Gersbach from Duke University in Durham, N.C., and his collaborators there and elsewhere. However, at this stage, that editing is directed to an exon 23 deletion in the mdx model of the disease in mice, with CRISPR/Cas 9-based editing changing “expression of a modified dystrophin gene in place of the defective one, [leading to] partial recovery of functional dystrophin protein in skeletal and cardiac muscle, improvement of muscle biochemistry, and significant enhancement of muscle force,” they report. “This is the first time CRISPR has been successfully delivered throughout the body with a translational approach to treat grown animals with genetic diseases,” points out Christopher Nelson, the lead author of their report that appeared 31 December 2015 in Science (doi:10.1126/science.aad5143).