Duchenne Muscular Dystrophy

Hereditary Disease - Duchenne Muscular Dystrophy (DMD) Disease, Symptoms, and Description of Survival Opportunities Duchenne Muscular Dystrophy is a hereditary disorder that causes one of 3,500 male voluntary muscles to weaken the movement of the body . It is wasted because there is no dystrophin. Dystrophin is an important muscle protein produced by the X chromosome gene. Therefore, it is a gender related disease affecting only boys. If you inherit the disease, the boy loses muscle in your life.

Duchenne muscular dystrophy was first described by the French neurologist Guillaume Benjamin Amand Duchenne in the 1860s, but it was until the 1980s that little was known about the cause of any type of muscular dystrophy. In 1986, researchers who were supported by MDA discovered specific genes that cause DMD on the X chromosome when it is defective (mutated). In 1987, a protein related to this gene was identified and named dystrophin. If muscle cells do not have dystrophin, the muscle cells become fragile and become susceptible to damage.

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

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 "and so on.