DiTullio P, Cheng S H, Marshall J, Gregory R J, Ebert K M, Meade H M, Smith A E
Genzyme Corporation, Framingham, Massachusetts 01701.
Biotechnology (N Y). 1992 Jan;10(1):74-7. doi: 10.1038/nbt0192-74.
Here we describe the production of cystic fibrosis transmembrane conductance regulator (CFTR), the product of the gene associated with cystic fibrosis, in the milk of transgenic mice. Mammary specific expression was achieved by placing the CFTR cDNA under the control of the goat beta-casein gene promoter. By fractionation, CFTR was shown to be associated with the membranes that envelop milk fat globules as they are discharged from the apical surface of the mammary epithelia. Since milk fat globules may comprise up to 10% of whole milk, this represents a novel, inexpensive and efficient approach to produce CFTR and possibly other membrane-associated proteins. The availability of large quantities of CFTR could have important implications for the development of new therapies for cystic fibrosis.
在此，我们描述了在转基因小鼠的乳汁中生产囊性纤维化跨膜传导调节因子（CFTR）的过程，CFTR是与囊性纤维化相关基因的产物。通过将CFTR cDNA置于山羊β-酪蛋白基因启动子的控制之下，实现了乳腺特异性表达。通过分级分离显示，CFTR与乳腺上皮细胞顶端表面排出的乳脂肪球周围的膜相关联。由于乳脂肪球可能占全脂牛奶的10%，这代表了一种生产CFTR以及可能其他膜相关蛋白的新颖、廉价且高效的方法。大量CFTR的可得性可能对囊性纤维化新疗法的开发具有重要意义。
