小鼠疾病模型,脊髓性肌肉萎缩症 z-bo 2020-10-19 696

　　A few days ago, Professor Hua Yimin of the Institute of Neuroscience of Soochow University and the Cold Spring Harbor Laboratory of the United States collaborated on a large number of mouse disease models and found that increasing the expression of motor neuron survivin in peripheral tissues can effectively treat spinal muscular atrophy. Related research results have been published in the internationally renowned biomedical journal Genes and Development. Hua Yimin is the first author and co-corresponding author.

　　Spinal muscular atrophy is a recessive autosomal inherited disease, and it is the number one killer of infant deaths. In the latest research, Hua Yimin and American collaborators designed a sense oligonucleotide called "bait" and injected it into the brain ventricle of mice, which disrupted the action of the drug in the central nervous system to ensure motor nerves. The increase in metasurvivin expression is limited to peripheral tissues. Surprisingly, this treatment approach can extend the life of mice by more than 20 times. This discovery breaks through the current view in this field, that is, it is believed that increasing the expression of motor neuron survivin in the spinal cord is a necessary condition for treating spinal muscular atrophy.