ReviewFrontiers in neuroscience2026
The delivery challenge of adeno-associated virus vector-based gene therapies for neurological diseases.
Review in Frontiers in neuroscience, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
2 citing papers in PubMed.
- Hypothalamic Control of Liver Health and Disease: From Circuits to Pathophysiology and Therapies.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Review
- Shifting From Systemic to Precision-Targeted Complement Therapies: Opportunities and Hurdles.European journal of immunology · 2026Review
Corrections and comments
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Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
There is great anticipation that gene therapies can offer solutions to many neurological diseases. Already, much is known about therapeutic targets and how they would need to be manipulated to mitigate disease. For such gene therapies to move to the clinic, potent CNS delivery vehicles are needed. One line of investigation focuses on adeno-associated viruses (AAV) to address this need. In particular, blood-brain barrier (BBB)-penetrant AAV capsids are of interest due to the relative ease of their intravenous administration. This review will introduce this topic and provide an update on recent developments. First, we describe the physical barriers that must be overcome for AAV-delivered gene therapies to reach target cells in the CNS. We then put a spotlight on the natural AAV9 capsid's inherent propensity to cross the BBB and key lessons learned from its use for delivering a therapeutic payload for the treatment of spinal muscular atrophy. Next, we summarize methods for engineering recombinant AAV (rAAV) capsids with improved brain penetrance, and present
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.