Comparative assessment of the transduction efficiency and safety associated with the delivery of AAV9-GFP vector via lumbar puncture to cynomolgus macaques with and without anti-AAV9 pre-existing antibodies

Comparative assessment of the transduction efficiency and safety associated with the delivery of AAV9-GFP vector via lumbar puncture to cynomolgus macaques with and without anti-AAV9 pre-existing antibodies

Administration of AAV-based gene therapies into the intra-cerebrospinal fluid (CSF) compartments via routes such as lumbar puncture (LP) has been implemented as an alternative to intravenous dosing to target the CNS regions. This route enables lower doses, decreases systemic toxicity, and circumvent...

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Bibliographic Details
Main Authors: Ghiabe H. Guibinga, Janet Do, Binh Chu, Yin Gu, Rie Kikkawa, Xiaoguang Li, Fatih Ozsolak, Timothy MacLachlan
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV9
intra-CSF dosing
pre-existing immunity
neutralizing antibody
toxicity
transduction efficiency
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050124001876
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