New gene correction therapy for Duchenne muscular dystrophy

Researchers at Technical University of Munich (TUM), Ludwig Maximilian University of Munich (LMU) and the German Research Center for Environmental Health (Helmholtz Zentrum München) have developed a gene therapy that may provide permanent relief for those suffering from DMD.

Muscles need dystrophin in order to regenerate.

Persons suffering from Duchenne muscular dystrophy lack this essential muscular protein due to mutations in the gene which is responsible for producing dystrophin.

DMD affects mainly boys, since the responsible mutations are located in the dystrophin gene on the X chromosome.

Gene scissors remove defective gene sequence

An interdisciplinary Munich research team led by scientists from TUM has for the first time succeeded in correcting the mutated dystrophin gene in living pigs.

"These gene scissors are highly efficient and specifically corrected the dystrophin gene," says Prof.

So if we change the genome of a myocardial cell, the correction is a long-term success, in contrast to the results of previous methods."

Therapeutic success with clinically relevant model

The gene sequence responsible for the dystrophin protein has already been successfully corrected in the past, however in mice and other animal models.