Gene Transfer to the CNS Using Recombinant Adeno-Associated Virus

Gene Transfer to the CNS Using Recombinant Adeno-Associated Virus

Gene Transfer to the CNS Using Recombinant Adeno-Associated Virus

ABSTRACT

Recombinant adeno-associated virus (rAAV) vectors are great tools for gene transfer due
to their ability to mediate long-term gene expression. rAAVs have been used successfully
as gene transfer vehicles in multiple animal models of CNS disorders, and several clinical
trials are currently underway. rAAV vectors have been used at various stages of development
with no apparent toxicity. There are multiple ways of delivering AAV vectors
to the mouse CNS, depending on the stage of development. In neonates, intravascular
injections into the facial vein are often used. In adults, direct injections into target regions
of the brain are achieved with great spatiotemporal control through stereotaxic surgeries.
Recently, discoveries of new AAV vectors with the ability to cross the blood brain barrier
have made it possible to target the adult CNS by intravascular injections.
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