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囊性纤维化(CF)是由于上皮细胞特异的氯离子通道——CF跨膜传导调节因子(CFTR)缺陷所引起的一种遗传性疾病。研究表明,利用基因治疗手段使CFTR在呼吸道上皮细胞表面表达,可部分改善其功能。其常用载体多为腺病毒,其中,对呼吸道上皮细胞有亲嗜性的2、5型腺病毒(Ad2、Ad5)更受青睐。然而,虽然该载体缺失了腺病毒的E_1序列,但并未完全消除腺病毒的其他早期及晚期基因的表达或阻止病毒的复制,从而
Cystic fibrosis (CF) is a genetic disorder caused by defects in the epithelial cell-specific chloride channel, the CF transmembrane conductance regulator (CFTR). Studies have shown that the use of gene therapy means that CFTR is expressed on the surface of respiratory epithelial cells and partially improves its function. The most common vectors are adenovirus, of which adenovirus type 2 and adenovirus 5 (Ad2, Ad5) are more favored for respiratory epithelial cells. However, although this vector lacks the E_1 adenovirus sequence, it does not completely abolish the expression of other early and late genes of the adenovirus or prevent the replication of the virus