Article

AAV2 Crosslinks Actin Filaments: Implications for AAV Gene Therapy Vector Design

2026-05-10

Abstract excerpt

Adeno-associated virus (AAV) capsids are important gene therapy vectors, allowing for the one-time treatment of monogenetic disorders, with durable gene expression lasting for years. However, despite the clinical success of AAV usage, low transduction efficiencies require high dosage to achieve therapeutic efficacy, resulting in prohibitive costs and rare, but life-threatening immune responses. One key knowledge g...

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Literature Corpus work: 542e14ec-579d-5f79-b7cc-388348207140
DOI: 10.64898/2026.05.08.723880

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