An adeno-associated virus vector penetrates the blood–brain barrier in non-human primates

Developing gene therapy for use in the central nervous system has been hampered by the lack of an efficient vector for gene delivery. We report an adeno-associated virus vector with an enhanced ability to cross the blood-brain barrier in both rodents and non-human primates, and use it to develop systemic anti-tumour gene therapies for glioblastoma.