Genzyme Corp has announced that its researchers have developed the first genetically-engineered mice to produce in their milk a membrane protein for possible therapeutic use in humans. The mice were developed at Tufts School of Veterinary Medicine, North Grafton, Massachusetts.
The discovery provides a means to produce large quantities of the membrane protein Cystic Fibrosis Transmembrane Regulator (CFTR), the protein coded by the gene associated with cystic fibrosis, as well as other therapeutic proteins. The research details were presented at the Miami Bio/Technology Winter Symposia last month and have since been published in the January issue of the journal Bio/Technology.
"These results are very exciting," said Alan Smith, Genzyme's senior vice president of research. He added: "first, this greatly extends the usefulness of production in transgenic milk to now include an important class of proteins that previously were considered difficult to make in large amounts. And second, further progress in seeking new therapies for cystic fibrosis and other genetic diseases will be facilitated by the availability of larger quantities of CFTR protein. When the technique is applied to larger animals, unlimited supplies of CFTR and other membrane proteins should become available."
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