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.
This is a preview of subscription content, access via your institution
Access options
Access Nature and 54 other Nature Portfolio journals
Get Nature+, our best-value online-access subscription
$29.99 / 30 days
cancel any time
Subscribe to this journal
Receive 12 digital issues and online access to articles
$99.00 per year
only $8.25 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
References
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.
Additional information
Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.
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).
Rights and permissions
About this article
Cite this article
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
Published:
Issue Date:
DOI: https://doi.org/10.1038/s41551-022-00939-6
