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Novel Engineered AAV Variants Demonstrate Superior Blood-Brain Barrier Penetration and Safety in Non-Human Primates

2026-04-01

Abstract excerpt

Systemic delivery of adeno-associated virus (AAV) for gene therapy of central nervous system (CNS) disorders is limited by inefficient blood-brain barrier (BBB) penetration and dose-limiting toxicity in peripheral organs, notably the liver and dorsal root ganglia (DRG) 1-5 . Here, we report the development of novel AAV variants via a proprietary capsid engineering platform (REACH). In non-human primates (NHPs), i...

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Literature Corpus work
a74aa7d7-332b-50a5-ae5a-c4442b609603
DOI
10.64898/2026.03.29.713052
Open publication

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Novel Engineered AAV Variants Demonstrate Superior Blood-Brain Barrier Penetration and Safety in Non-Human PrimatesDOI 10.64898/2026.03.29.713052
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