Thetreatmentofmusculardystrophy.pptVIP

The treatment of muscular dystrophy 1 The treatment of pharmaceuticals 2 Gene therapy 3 Cell transplantation therapy [Mesenchymal Stem Cell Transplant to Treat Duchenne Muscular Dystrophy ] The mdx mouse——肌营养不良实验鼠 These dystrophic mice have a point mutation that exchanged the base cytosine C in position 3,185 in exon 23 against a thymine T . This converted the CAA codon, which normally codes for the amino acid glutamine, to the triplet TAA in the mRNA it become UAA which is a premature stop codon. 它是一种点突变实验鼠，基因突变发生在肌营养不良蛋白23号外显子的第3185号核苷酸上，将本该编码为谷氨酸盐氨基酸的CAA码，改变成了TAA码，这是一个停止合成蛋白