Whitsett J A, Dey C R, Stripp B R, Wikenheiser K A, Clark J C, Wert S E, Gregory R J, Smith A E, Cohn J A, Wilson J M
Division of Pulmonary Biology, Children's Hospital, Cincinnati, Ohio 45229-2899.
Nat Genet. 1992 Sep;2(1):13-20. doi: 10.1038/ng0992-13.
Human cystic fibrosis transmembrane conductance regulator (CFTR) was expressed in transgenic mice under the control of transcriptional elements derived from the human surfactant protein C (SP-C) gene. The hCFTR mRNA was expressed in lungs and testes: in the lung, we found hCFTR mRNA in bronchiolar and alveolar epithelial cells, and CFTR protein in respiratory epithelial cells. While the level of expression of hCFTR mRNA varied, hCFTR mRNA and protein were detected in pulmonary epithelial cells of several lines. Lung weight, morphology, somatic growth and reproductive capacity were not altered by expression hCFTR in lung and testes of the transgenics. Our findings suggest that hCFTR can be safely transferred to lung epithelial cells for CF therapy.
人类囊性纤维化跨膜传导调节因子（CFTR）在源自人类表面活性蛋白C（SP-C）基因的转录元件控制下在转基因小鼠中表达。hCFTR mRNA在肺和睾丸中表达：在肺中，我们在细支气管和肺泡上皮细胞中发现了hCFTR mRNA，在呼吸道上皮细胞中发现了CFTR蛋白。虽然hCFTR mRNA的表达水平有所不同，但在多个品系的肺上皮细胞中都检测到了hCFTR mRNA和蛋白。转基因小鼠肺和睾丸中hCFTR的表达并未改变肺重量、形态、体细胞生长和生殖能力。我们的研究结果表明，hCFTR可以安全地转移到肺上皮细胞用于囊性纤维化治疗。
