Duchenne muscular dystrophy (DMD) is a recessive X-linked form of muscular dystrophy. It affects around 1 in 3,600 boys. In this disease there is a progressive loss of muscle function and weakness, which begins in the lower limbs. Over time, patients with the disorder suffer gradual muscle deterioration, which leads to paralysis and eventual death, usually by age 25.
The disorder is caused by a mutation in the dystrophin gene, the largest gene located on the human X chromosome, which codes for the protein dystrophin, an important structural component within muscle tissue that provides structural stability to the dystroglycan complex (DGC) of the cell membrane.
Bio-medical engineers of Duke University by using genetic editing technique, have been able to repair a defect responsible for Duchenne muscular dystrophy, in cell samples from the patients.
The bio-medical engineers have developed a way to change the existing mutated gene responsible for the disorder into a normally functioning gene instead of, gene therapy approach of adding new genetic material to "override" the faulty gene. They believe that their approach could be safer and more stable than current methods of gene therapy.
The researchers are now conducting further tests of this new approach in animal models of the disease.
The results of the Duke study were published online in Molecular Therapy, the journal of the American Society for Gene and Cell Therapy.
For their experiment they used synthetic proteins known as transcription activator-like effector nucleases (TALENs), which are artificial enzymes that can be engineered to bind to and modify almost any gene sequence.The TALEN finds its target site in the human genome by binding to DNAwith an engineered DNA-recognition protein. Once the protein finds its target site, the DNA is modified by the enzyme domain of the protein
Duchenne muscular dystrophy has been extensively studied by scientists, and it is believed that more than 60 percent of patients with this type of mutation can be treated with this novel genetic approach. The approach could be helpful in treating other genetic diseases where a few gene mutations.
The disorder is caused by a mutation in the dystrophin gene, the largest gene located on the human X chromosome, which codes for the protein dystrophin, an important structural component within muscle tissue that provides structural stability to the dystroglycan complex (DGC) of the cell membrane.
Bio-medical engineers of Duke University by using genetic editing technique, have been able to repair a defect responsible for Duchenne muscular dystrophy, in cell samples from the patients.
The bio-medical engineers have developed a way to change the existing mutated gene responsible for the disorder into a normally functioning gene instead of, gene therapy approach of adding new genetic material to "override" the faulty gene. They believe that their approach could be safer and more stable than current methods of gene therapy.
The researchers are now conducting further tests of this new approach in animal models of the disease.
The results of the Duke study were published online in Molecular Therapy, the journal of the American Society for Gene and Cell Therapy.
For their experiment they used synthetic proteins known as transcription activator-like effector nucleases (TALENs), which are artificial enzymes that can be engineered to bind to and modify almost any gene sequence.The TALEN finds its target site in the human genome by binding to DNAwith an engineered DNA-recognition protein. Once the protein finds its target site, the DNA is modified by the enzyme domain of the protein
Duchenne muscular dystrophy has been extensively studied by scientists, and it is believed that more than 60 percent of patients with this type of mutation can be treated with this novel genetic approach. The approach could be helpful in treating other genetic diseases where a few gene mutations.
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