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Article

Comparison of AAV9-driven motor neuron transduction following different CNS-directed delivery methods in mice

2025-07-03

Abstract excerpt

<h4> Abstract </h4> <h4>Background</h4> Gene therapies are promising for diseases previously considered incurable. Adeno-associated virus serotype 9 (AAV9) demonstrates remarkable tropism for motor neurons (MNs) and represents an exciting candidate to target genetic causes of motor neuron diseases like amyotrophic lateral sclerosis (ALS). However, systemic delivery risks immunogenicity and off-target effects, t...

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Literature Corpus work
0a66341f-2726-53b6-9a11-3922dd88cfe5
DOI
10.1101/2025.07.01.662642
Open publication

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Comparison of AAV9-driven motor neuron transduction following different CNS-directed delivery methods in miceDOI 10.1101/2025.07.01.662642
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