Muscular Dystrophy

Guillaume B.A. was discovered in 1861. Duchenne, muscular dystrophy is a very regrettable disease. Muscular dystrophy is a group of hereditary disorders characterized by progressive muscle weakness and exhaustion. Features of muscular dystrophy may be transmitted as autosomal dominant. In this case, children may have 55 genetic diseases if there are children in some of the original carriers of the disease. It is also carried as an autosomal recessive trait, in which case the offspring of the original carrier will have a small possibility of genetic trait.

There are various kinds of muscular dystrophy. For example, Duchenne muscular dystrophy, Becker malnutrition, Emery-Dreyfus muscular dystrophy, myotonic dystrophy, limb grid type malnutrition, facial cranial brachial muscular dystrophy, congenital malnutrition, ocular malnutrition and distal muscular dystrophy. Duchenne's malnutrition occurs in 2 out of 10,000 young men. It is the most serious disease of all muscular dystrophies. It mainly occurs in young boys affecting children today. Symptoms and signs of muscular dystrophy include sudden falls, muscles of the calf thighs, difficulty going out when lying or sitting, difficulties in running and jumping, weakness in the muscles of the feet, Mental retardation "etc.

Disease muscular dystrophy is a disease characterized by weakness and deterioration of skeletal muscle. There are more than 30 different forms of muscular dystrophy, but the two most common are Duchenne and Becker muscular dystrophy (Baronceri). These two forms of disease are sexually related, and mainly affect men. Men usually do not accept other X chromosomes to inherit the defective X chromosome from the mother and neutralize the defective X chromosome (male is XY). Affected people have difficulty in performance

Duchenne Muscular Dystrophy (DMD): DMD is the most common form of muscular dystrophy. This is because the dystrophin gene mutation is on the X chromosome, mainly because it affects boys. This is the most common form of muscular dystrophy and is one of the most common genetic diseases of humans, and it occurs in boys of approximately 1: 5000. The boy lost his ability to walk between teens and died in the early days of adulthood. Dystrophin is the largest gene in the human genome (the 11 kb coding region) and is therefore too large to be packaged in an AAV vector. Micro-muscular dystrophy proteins, including the most important regions of the gene but excluding redundant aspects, have been developed for AAV gene therapy. Approximately 40% of the micro-muscular dystrophy protein representing the coding sequence of the human dystrophin gene was delivered to the biceps brachii of six boys by intramuscular injection using rAAV 2.5. Micro-muscular dystrophy was detected in 2 out of 4 cases at 42 days but not in 2 cases 90 days after injection