Stem cell transplantation for progressive muscular dystrophy 1.What is called progressive muscular dystrophy? Progressive myotonic dystrophy is a hereditary progressive muscle degeneration disease, mainly manifested by progressive muscle atrophy and weakness, in recent decades, due to the application of electron microscopy technology, genetic genes, biochemistry, histochemistry, enzymology and other research progresses rapidly, the pathological research on myotonic dystrophy is gradually centered on pseudohypertrophic myotonic dystrophy (Duchenne) The disease can be broadly classified into different genetic patterns. Due to the different genetic patterns, they can be broadly classified as follows: (1): Duchenne type with X chromosome recessive inheritance and benign pseudohypertrophic muscular dystrophy (Becker); (2): autosomal dominant inheritance: facial —- shoulder —- brachial type; (3): autosomal recessive inheritance of limb-girdle type, etc. Most of them start in adolescence, some later, and the first symptom is the weakness and atrophy of pelvic girdle muscle, and the disease progresses slowly and gradually involves the scapular girdle and the difficulty of raising both arms, while the advanced patients may have muscle contracture and cannot move without intellectual impairment, and the severity and progress speed of the disease varies greatly, and generally does not affect life expectancy. Among the various types of myotonic dystrophy, the Duchenne type has the highest incidence and severity, and is the most representative disease of hereditary myotonic dystrophy. The typical duck-step shape when walking and the difficulty in ascending and walking is another characteristic of the disease. When squatting, the patient can’t stand up or has difficulty standing up after squatting. If not properly treated often early disability and lead to death. 2, what are the conventional treatment methods of progressive muscular dystrophy? For myotonic dystrophy, all Chinese and Western medicines have no long-term effect. Myotonic dystrophy patients should participate in daily work and strength training to prevent joint contracture. Oculomotor dystrophy can be treated surgically, scoliosis can be surgically orthopedic, and it is uncertain whether subcutaneous Achilles tendon surgery for joint contracture can prolong the patient’s walking time. In the later stage, special attention should be paid to the patient’s respiratory function to improve lung capacity and respiratory muscle capacity through respiratory exercises and assisted breathing. In addition a high protein diet should be administered.
At present, the focus should be on blocking the disease inheritance through family planning method, and prenatal genetic diagnosis is only a remedy. 3.How does stem cell transplantation treat progressive muscular dystrophy? The culprit of muscular dystrophy is a gene called dystrophin. Both deletion and mutation of this gene may lead to muscular dystrophy. Stem cells can promote muscle regeneration and provide dystrophin, a dystrophin deficient protein, to the damaged muscles of muscular dystrophy, thus achieving the purpose of treatment and dystrophy.