小鼠肌肉萎缩症,动物造模 z-bio 2021-10-09 549

　　For the first time, researchers used gene editing technology to successfully reverse the degenerative disease in mice. This technology will help treat similar human diseases in the future.

　　Three research papers were published in the US "Science" weekly magazine. Researchers applied CRISPR (clustered, regularly spaced short-interval palindromic repetition) technology to adult mice with pseudohypertrophic muscular dystrophy. Currently, 1 in 5,000 boys worldwide suffers from this disease.

　　researchers said: "This is the first time that CRISPR technology has successfully treated adult mammalian genetic diseases. This technology is expected to replace human diseases."

　　Pseudohypertrophic muscular dystrophy weakens the patient’s muscles. It is possible. This is because genetic variation prevents muscle cells from normally producing a protein called dystrophin, which is necessary for normal muscle formation.

　　The new gene editing technology can repair the gene mutation that causes the disease. This method uses non-pathogenic viruses as the "carrying pathway" of artificially modified genes to make accurate changes to the DNA of diseased mice.

　　Charles Gersbach, an associate professor of biomedical engineering at Duke University, is the head of the research paper. After perfecting the CRISPR technology, Gersbach and his team applied this method directly to the leg muscle treatment of adult mice for the first time. As a result, the muscle strength of the mouse was improved, and the condition of other parts of the mouse, such as the heart muscle, was improved.