Abstract
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. These new reports complement recent articles published in MT and elsewhere suggesting that AAV9 may soon take its place alongside AAV2 in the field of AAV-based gene therapy.
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