Systemic gene delivery to the central nervous system using Adeno-associated virus.

Mathieu Bourdenx, Nathalie Dutheil, Erwan Bezard, Benjamin Dehay
Front. Mol. Neurosci.. 2014-06-02; 7:
DOI: 10.3389/fnmol.2014.00050

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1. Front Mol Neurosci. 2014 Jun 2;7:50. doi: 10.3389/fnmol.2014.00050. eCollection

Systemic gene delivery to the central nervous system using Adeno-associated

Bourdenx M(1), Dutheil N(1), Bezard E(1), Dehay B(1).

Author information:
(1)Institut des Maladies Neurodégénératives, UMR 5293, Université de Bordeaux
Bordeaux, France ; CNRS, Institut des Maladies Neurodégénératives, UMR 5293
Bordeaux, France.

Adeno-associated virus (AAV)-mediated gene delivery has emerged as an effective
and safe tool for both preclinical and clinical studies of neurological
disorders. The recent discovery that several serotypes are able to cross the
blood-brain barrier when administered systemically has been a real breakthrough
in the field of neurodegenerative diseases. Widespread transgene expression after
systemic injection could spark interest as a therapeutic approach. Such strategy
will avoid invasive brain surgery and allow non-focal gene therapy promising for
CNS diseases affecting large portion of the brain. Here, we will review the
recent results achieved through different systemic routes of injection generated
in the last decade using systemic AAV-mediated delivery and propose a brief
assessment of their values. In particular, we emphasize how the methods used for
virus engineering could improve brain transduction after peripheral delivery.

DOI: 10.3389/fnmol.2014.00050
PMCID: PMC4040820
PMID: 24917785

Auteurs Bordeaux Neurocampus