囊性纤维化基因治疗的临床前和临床终点检测

Pre-clinical and clinical endpoint assays for cystic fibrosis gene therapy.

作者信息

Griesenbach Uta, Boyd A Christopher

机构信息

Department of Gene Therapy, Faculty of Medicine, Imperial College London, UK.

出版信息

J Cyst Fibros. 2005 May;4(2):89-100. doi: 10.1016/j.jcf.2005.02.002.

DOI:10.1016/j.jcf.2005.02.002

PMID:15914096

Abstract

The credibility and hence value of pre-clinical and clinical cystic fibrosis gene therapy studies depend on the assays used to evaluate gene transfer. Awareness of assay suitability, sensitivity and variability is therefore crucial to the design of experimental programmes. Here, we review the assays that are in use to assess the efficacy of gene transfer in pre-clinical and clinical CF gene therapy research, highlight their weaknesses and suggest possible new strategies that may help to overcome current limitations.

摘要

临床前和临床囊性纤维化基因治疗研究的可信度以及由此产生的价值取决于用于评估基因转移的检测方法。因此，了解检测方法的适用性、灵敏度和变异性对于实验方案的设计至关重要。在此，我们综述了目前用于评估临床前和临床囊性纤维化基因治疗研究中基因转移效果的检测方法，强调了它们的不足之处，并提出了可能有助于克服当前局限性的新策略。