Optimized AAV capsids for basal ganglia diseases show robust potency and distribution

Optimized AAV capsids for basal ganglia diseases show robust potency and distribution

Abstract Huntington’s disease and other disorders of the basal ganglia create challenges for biomolecule-based medicines given the poor accessibility of these deep brain structures following intracerebral or intravascular delivery. Here, we found that low dose, low volume delivery of unbiased AAV li...

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Main Authors: D. E. Leib, Y. H. Chen, L. Tecedor, P. T. Ranum, M. S. Keiser, B. C. Lewandowski, E. M. Carrell, S. Arora, I. Huerta-Ocampo, D. Lai, C. M. Fluta, C. Cheng, X. Liu, B. L. Davidson
Format: Article
Language:English
Published: Nature Portfolio 2025-05-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-60000-3
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https://doi.org/10.1038/s41467-025-60000-3

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