ArticleCell reports2025
AAVs targeting human carbonic anhydrase IV enhance gene delivery to the brain.
Article in Cell reports, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
What it found
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The trial behind it
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Who cites it
5 citing papers in PubMed.
- Breaking Biological Barriers: Engineering Nanocarriers for Efficient Drug and Gene Delivery through Nano-Bio Interfaces and Biophysical Design.Applied physics reviews · 2026Article
- CA-IV-directed small-molecule shuttle enables targeted brain delivery of biologics.Nature chemical biology · 2026Article
- Defining RNA oligonucleotides that reverse deleterious phase transitions of RNA-binding proteins with prion-like domains.Molecular cell · 2026Article
- The delivery challenge of adeno-associated virus vector-based gene therapies for neurological diseases.Frontiers in neuroscience · 2026Review
- Species barriers in AAV tropism: mechanisms, models, and emerging solutions for clinical translation.Acta biochimica Polonica · 2026Review
Corrections and comments
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Authors and funding
15 authors.
Funding
Abstract
Gene therapies using natural adeno-associated virus (AAV) serotypes have restricted applications, particularly in the brain, due to their poor targeting and resulting safety concerns. Directed evolution has identified brain-enhanced engineered AAV capsids for various model organisms, but inter-species differences challenge their translation. Here, we engineer AAVs to target human carbonic anhydrase IV (CA-IV), a recently identified blood-brain barrier transcytosis receptor with several favorable properties. We perform in vitro on-column selection of an AAV library to exclude capsids that do not target human CA-IV. We subsequently screen the top 0.01% of variants in vivo in mice expressing human CA-IV in brain endothelial cells. Notably, two top-performing capsids in "humanized" mice are relatively lower ranked in vitro and outperform several top-ranked in vitro candidates. One of these, AAV-hCA4-IV77, achieves 100-fold-greater brain transduction than AAV9, with robust neuronal and astrocytic coverage across brain regions. These results advance our understanding of receptor-targeted capsid design and support the therapeutic potential of human CA-IV-engaging AAVs.
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Registered trials
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.