One of the long-term aims of gene therapy is to deliver vectors to diseased organs directly from the circulation. Efforts toward this end with recombinant adeno-associated virus (AAV)-based vectors have been hampered by the fact that most of the vector ends up in the liver after systemic delivery. In addition, tissue-specific barriers such as the bloodbrain barrier (BBB) can limit the entry of vectors from the blood to certain organs. Two papers in this issue of Molecular Therapy describe progress in addressing these obstacles with respect to a relative newcomer to the family of AAV vector serotypes, AAV9.
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Forsayeth et al. (2011) studied this question.
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