Advances in AAV-mediated gene transfer for the treatment of inherited disorders.

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.