Journal
EUROPEAN JOURNAL OF HUMAN GENETICS
Volume 12, Issue 4, Pages 263-271Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/sj.ejhg.5201153
Keywords
adeno-associated viral vectors; gene therapy; inherited disorders
Funding
- NEI NIH HHS [1R01EY015136-01, R01 EY015136] Funding Source: Medline
- Telethon [TGM06S01, TGM06S02] Funding Source: Medline
Ask authors/readers for more resources
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.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available