中国临床药理学与治疗学2008，Vol.13Issue(10)：1182-1194,13.
基因治疗用腺相关病毒载体的剂量效应与剂量控制
Dose-response and control of adeno-associated viral vectors based preclinical and clinical gene therapy
摘要
Abstract
Human gene therapy needs to express exogenous DNA at the targeting cells,producing a practical and efficient therapeutic dosage at an approp-riate time(quantitative pharmacology)with a safe man-ner.Recombinant adeno-associated virus(rAAV)Vec-tom possess a number of properties and recent progress in rAAV production made it rapidly become the reagent of choice for therapeutic gene tmasfer.Over 60 clinical trials of gene therapy based on rAAV have been carried out.The dose response reaction between rAAV vectors and gene expression activity or clinical outcome is one of major aspects of these trials.Most studies showed that vector genomes(vg)and gene expression had a concentration-dependent relationship during a certain scope.However,gene expr~sion Can be afffected by viral serotypes,tissue tropisms,cell targeting,drug regulation,injection route,age and sex,etc.Thus,these aspects should be carefully comidered by scienti-sts,pharmacologisis and physicians during animal ex-periments or clinical trails.KEY WORDS gene therapy;viral vector;dose-re-sponse;quantitative pharmacology;clinical thempy关键词
基因治疗/病毒载体/剂量效应/定量药理学/临床治疗分类
医药卫生引用本文复制引用
王启钊,吕颖慧,许瑞安..基因治疗用腺相关病毒载体的剂量效应与剂量控制[J].中国临床药理学与治疗学,2008,13(10):1182-1194,13.基金项目
Project supported by 863 national pmjects(2007AA022194),Xiammen projects(3502Z20083046) （2007AA022194）
