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.
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Mendell, J. R. et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N. Engl. J. Med. 377, 1713–1722 (2017). An article that presents the first clinical success of AAV9-mediated gene therapy.
Hudry, E. & Vandenberghe, L. H. Therapeutic AAV gene transfer to the nervous system: a clinical reality. Neuron 101, 839–862 (2019). A review article that presents the progress and challenges of CNS gene delivery using AAV.
Gray, S. J. The evolution of adeno-associated virus capsids for CNS gene therapy. Cell Gene Ther. Insights 5, 1361–1368 (2019). A review article that presents the limitations of current AAV capsids for CNS application.
Sarkaria, J. N. et al. Is the blood-brain barrier really disrupted in all glioblastomas? A critical assessment of existing clinical data. Neuro. Oncol. 20, 184–191 (2018). This review article presents emerging clinical evidence of the critical role of the BBB in glioblastoma treatment.
Goertsen, D. et al. AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset. Nat. Neurosci. 25, 106–115 (2021). Using a directed revolution screening method, this article identifies AAV capsids that penetrate the BBB in mice and marmosets.
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This is a summary of: Yao, Y. et al. Variants of the adeno-associated virus serotype 9 with enhanced penetration of the blood–brain barrier in rodents and primates. Nat. Biomed. Eng. https://doi.org/10.1038/s41551-022-00938-7 (2022).
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An adeno-associated virus vector penetrates the blood–brain barrier in non-human primates. Nat. Biomed. Eng 6, 1201–1202 (2022). https://doi.org/10.1038/s41551-022-00939-6