Article

Optimized AAV capsids for diseases of the basal ganglia show robust potency and distribution in adult nonhuman primates

2024-05-05

Abstract excerpt

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. Additionally, for adeno-associated viruses (AAVs) intravascular delivery exposes peripheral tissues to the vast majority of the therapy, increasing the risk of immune responses and the quantity...

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Literature Corpus work: 05e24cde-876d-50ab-94db-e820a004c003
DOI: 10.1101/2024.05.02.592211

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