Gene therapy can provide permanent relief for patients with DMD

Duchenne muscular dystrophy (DMD) is the most common inherited muscle disease in children In response, researchers at institutions such as the University of technology in Munich (tum) have developed a gene therapy that can provide permanent relief for patients with DMD Dystrophin is the key to muscle regeneration People with DMD lack the necessary protein due to gene mutation, so their muscle cells will degenerate over time and gradually be replaced by connective tissue and adipose tissue DMD mainly affects men because the key mutation is on the X chromosome Through optimizing the editing technology of crispr-cas9 gene, the scientists of tum successfully repaired the mutated dystrophin gene in pigs for the first time Professor Wolfgang wurst, a geneticist at tum and the German Center for environmental health research, said: "these gene scissors are very effective and can correct the dystrophin gene." Compared with the animals without the treatment, the treated animals were less sensitive to arrhythmia and had increased life expectancy Professor Christian kupatt, a cardiologist, said: "muscle and heart cells are the cellular structure of longevity Half of all cardiomyocytes remain functional throughout the life cycle from birth to death "As long as the cell is alive, the genome of the cell can be used for protein biosynthesis Once the cell is affected by the treatment, it will remain in a corrected state Therefore, if we change the genome of cardiomyocytes, we will achieve long-term results " Source of information: new generation correction therapy for Duchenne miscellaneous dystrophy