期刊
EUROPEAN JOURNAL OF HUMAN GENETICS
卷 12, 期 4, 页码 263-271出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/sj.ejhg.5201153
关键词
adeno-associated viral vectors; gene therapy; inherited disorders
资金
- NEI NIH HHS [1R01EY015136-01, R01 EY015136] Funding Source: Medline
- Telethon [TGM06S01, TGM06S02] Funding Source: Medline
The holy grail of gene therapy is the cure of genetic diseases. To achieve this goal, a vector system is desirable that offers a high level of safety combined with clinical efficacy and versatility in terms of potential applications. Gene therapy vectors based on recombinant adeno-associated viruses (AAVs) meet all of these criteria: They are nonpathogenic, devoid of viral coding sequences, and mediate long-term gene expression in the absence of an immune or inflammatory response. Moreover, with the recent discovery of novel AAV serotypes, there is now one preferred serotype for nearly every organ or tissue to target. Thus, AAV gene therapy vectors are increasingly becoming the vectors of choice for the treatment of inherited disorders.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据