ArticleMolecular therapy : the journal of the American Society of Gene Therapy2026
Focused ultrasound expands intra-CSF AAV delivery to deep brain regions in rats and non-human primates.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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Who cites it
2 citing papers in PubMed.
- Focused Ultrasound is Poised to Take Non-Viral Gene Therapies to the Blood-Brain Barrier - and Beyond.Cell biomaterials · 2026Article
- Noninvasive Focal Gene Delivery into the Cerebellum of Non-Human Primates using Focused Ultrasound.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
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Authors and funding
21 authors.
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Abstract
A single administration of recombinant adeno-associated viruses (AAVs) has the ability to provide long-term gene expression in the brain, offering promising therapeutic options for neurodegenerative diseases that require decades of intervention. Intra-cerebrospinal fluid (CSF) administration is clinically relevant, and it enables broad transduction of superficial brain areas at lower doses compared with systemic delivery. However, intra-CSF AAV administration remains ineffective at transducing subcortical structures such as the striatum. We demonstrate in rats that following intra-CSF delivery of AAVs, targeting the striatum with focused ultrasound in the presence of intravenous microbubbles significantly enhances transduction in this brain region. When scaled to non-human primates, the AAV delivery to the caudate and putamen was increased up to 100-fold, and transgene expression up to 500-fold. These findings support intra-CSF administration of AAV combined with focused ultrasound as a promising strategy to extend gene delivery from superficial to deep brain structures with anatomical precision and clinical scalability.
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