Article

AAVs Targeting Human Carbonic Anhydrase IV Enhance Gene Delivery to the Brain

2025-04-22

Abstract excerpt

<h4>Summary</h4> Clinically approved gene therapies based on natural adeno-associated virus (AAV) serotypes have restricted applications, particularly in the brain, due to their poor targeting, high dose requirements, and resulting safety concerns. Directed evolution of enhanced AAV capsids in mice or non-human primates (NHPs) has resulted in markedly improved performance in those species, but inter-species diffe...

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Literature Corpus work: b691eb40-099d-5f0f-9200-425f08e2a7c7
DOI: 10.1101/2025.04.21.649868

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