摘要
重组腺相关病毒载体(rAAV)基因药物已经开展六十余项(67)临床研究,其安全、高效、稳定、表达持久等特点越来越受到业界的重视,最近的临床试验发现其在治疗先天性黑内障临床研究中呈现出显著疗效更是极大地振奋了人们的信心。临床研究案例的增加使人们对rAAV基因药物有了更为全面、深入的认识。与此同时,也对基因药物提出了更多挑战与要求,尤其是免疫原性和安全性等方面。
Numerous clinical trials (67) are using adeno-associated vectors (rAAV) as a gene delivery system so far.It becomes more and more alluring by its safety,efficiency,stability and long expression profiles.Recently,exciting outcomes have been obtained by rAAV clinical application in a retinal degeneration disease,Leber's congenital amaurosis.Clinical trails provide better information for us to understand about the rAAV based gene drugs and bring more challenges as well,immunogenicity and drug safety,etc,in particular.
出处
《中国生物工程杂志》
CAS
CSCD
北大核心
2010年第1期73-79,共7页
China Biotechnology
基金
国家“863”计划(2008AA02Z135)
国家重大新药创制专项(2009ZX09103-643)
华侨大学校资金(06Y0045
09BS517)资助项目