Thanks to the CRISPR-Cas9 method, the testing of stem cells and genome printing can be used to introduce promising gene therapy for Duchenne Muscular Dystrophy. This neuromuscular disease affects 1 boy out of 3,500 births, i.e. one birth every 3 days in France. 30,000 people are affected by this disease world-wide.
Already tested in mice, the approach uses the famous genome printing technology, namely CRISPR-cas9, already tested by a team from the UT Southwestern Medical Center “to prevent the advance of a form of muscular dystrophy comparable to Duchenne muscular dystrophy”. According to Dr. Spencer, Professor of Neurology and Co-Director of the Muscular Dystrophy Center at UCLA, “the new approach described here should be operational in clinical practice in approximately 10 years”.
Between now and then, “other animal tests should be carried out thoroughly prior to clinical trials in humans with the aim of implementing a method that can be used in humans in clinical practice”.