Article

Comparative Study of BBB-Targeting AAV Capsids on Central Nervous System Delivery Efficiency

2025-12-25

Abstract excerpt

The efficacy of adeno-associated virus (AAV)-mediated systemic gene therapy for central nervous system (CNS) diseases is often limited by the blood-brain barrier (BBB). This study systematically evaluated the tissue distribution of three BBB-crossing AAV capsid variants (PHP.eB, CNSRCV300, and BI-hTFR1) following intravenous injection in mice, using either a constitutive promoter (CAG) or a neuron-specific promote...

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Literature Corpus work: b38e3f95-d5ff-50c9-9fe9-17fda461f7d5
DOI: 10.64898/2025.12.23.696327

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