Absence of an intrathecal immune reaction to a helper-dependent adenoviral vector delivered into the cerebrospinal fluid of non-human primates

E. Butti, A. Bergami, A. Recchia, E. Brambilla, D. Franciotta, A. Cattalini, A. Stornaiuolo, F. Lachapelle, G. Comi, F. Mavilio, G. Martino, R. Furlan

Research output: Contribution to journalArticlepeer-review

Abstract

Inflammation and immune reaction, or pre-existing immunity towards commonly used viral vectors for gene therapy severely impair long-term gene expression in the central nervous system (CNS), impeding the possibility to repeat the therapeutic intervention. Here, we show that injection of a helper-dependent adenoviral (HD-Ad) vector by lumbar puncture into the cerebrospinal fluid (CSF) of non-human primates allows long-term (three months) infection of neuroepithelial cells, also in monkeys bearing a pre-existing anti-adenoviral immunity. Intrathecal injection of the HD-Ad vector was not associated with any sign of systemic or local toxicity, nor by signs of a CNS-specific immune reaction towards the HD-Ad vector. Injection of HD-Ad vectors into the CSF circulation may thus represent a valuable approach for CNS gene therapy allowing for long-term expression and re-administration.

Original languageEnglish
Pages (from-to)233-238
Number of pages6
JournalGene Therapy
Volume15
Issue number3
DOIs
Publication statusPublished - Feb 2008

ASJC Scopus subject areas

  • Genetics

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