Article

New AAV9 engineered variants with enhanced neurotropism and reduced liver off-targeting in mice and marmosets

2023-06-28

Abstract excerpt

<h4>Summary</h4> Adeno-Associated Virus 9 (AAV9) is a delivery platform highly exploited to develop gene-based treatments for neurological disorders given its low pathogenicity and brain tissue tropism. However, the efficacy of this vector is dampened by its relatively low efficiency to cross the adult blood-brain barrier (BBB) and inherent targeting to the liver upon intravenous delivery. We generated a new pept...

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Literature Corpus work: 26760a88-45b4-5053-b828-3db501f59582
DOI: 10.1101/2023.06.27.546696

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