Hope for Duchenne

Professor Tremblay's team partnered with Cellectis, a French firm specializing in genome engineering, in order to design enzymes-called meganucleases-with the ability to correct the dystrophin gene. During in vitro testing, the researchers inserted genes coding for a variety of meganucleases into human muscle cells.

They repeated the experiment in vivo with mice carrying the mutation that causes the illness. Both series of testing showed that the meganucleases can lead to a restoration of the normal nucleotide sequences of the dystrophin gene and its expression in muscle cells.