Flotte T R
Eudowood Division of Pediatric Respiratory Sciences, Johns Hopkins University School of Medicine, Johns Hopkins Hospital, Baltimore, Maryland 21205.
J Bioenerg Biomembr. 1993 Feb;25(1):37-42. doi: 10.1007/BF00768066.
Gene therapy for cystic fibrosis (CF) could potentially be accomplished with one of several recombinant virus vectors, including a murine retrovirus (MMuLV), adenovirus, or adeno-associated virus (AAV). All these vectors take advantage of their respective viruses' mechanisms for delivery of viral DNA to cells, evasion of lysosomal degradation, and optimization of the levels and duration of expression of viral (or vector) DNA. Each has its own unique life cycle, however. The differences among these viruses result in certain advantages and disadvantages, such as the requirement of retroviruses for active cell division, and the potential pathogenic effects from expression of certain adenovirus genes present in adenovectors. While no single vector may be optimal for CF gene therapy in humans, new techniques, such as receptor-mediated gene transfer, seek to take advantage of the desirable properties of one or more of the virus-based systems while avoiding certain potential hazards.
囊性纤维化（CF）的基因治疗可以通过几种重组病毒载体之一来实现，包括鼠逆转录病毒（MMuLV）、腺病毒或腺相关病毒（AAV）。所有这些载体都利用其各自病毒的机制将病毒DNA递送至细胞、逃避溶酶体降解以及优化病毒（或载体）DNA的表达水平和持续时间。然而，每种载体都有其独特的生命周期。这些病毒之间的差异导致了某些优点和缺点，例如逆转录病毒需要活跃的细胞分裂，以及腺载体中存在的某些腺病毒基因表达可能产生的致病效应。虽然没有一种载体可能对人类CF基因治疗是最佳的，但新技术，如受体介导的基因转移，试图利用一种或多种基于病毒的系统的理想特性，同时避免某些潜在风险。
