Journal
NATURE REVIEWS GENETICS
Volume 12, Issue 5, Pages 341-355Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nrg2988
Keywords
-
Categories
Funding
- Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia
- Howard Hughes Medical Institute
- US National Institutes of Health [HL64190, HL078810]
Ask authors/readers for more resources
In vivo gene replacement for the treatment of inherited disease is one of the most compelling concepts in modern medicine. Adeno-associated virus (AAV) vectors have been extensively used for this purpose and have shown therapeutic efficacy in a range of animal models. Successful translation to the clinic was initially slow, but long-term expression of donated genes at therapeutic levels has now been achieved in patients with inherited retinal disorders and haemophilia B. Recent exciting results have raised hopes for the treatment of many other diseases. As we discuss here, the prospects and challenges for AAV gene therapy are to a large extent dependent on the target tissue and the specific disease.
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