Efficient gene transfer into neurons in monkey brain by adeno-associated virus 8.
Although the adeno-associated virus (AAV) vector is a promising tool for gene transfer into neurons, especially for therapeutic purposes, neurotropism in primate brains is not fully elucidated for specific AAV serotypes. Here, we injected AAV serotype 8 (AAV8) vector carrying the enhanced green fluorescent protein (EGFP) gene under a ubiquitous promoter into the cerebral cortex, striatum and substantia nigra of common marmosets. Robust neuronal EGFP expression was observed at all injected sites. Cell typing with immunohistochemistry confirmed efficient AAV8-mediated gene transfer into the pyramidal neurons in the cortex, calbindin-positive medium spiny neurons in the striatum and dopaminergic neurons in the substantia nigra. The results indicate a preferential tropism of AAV8 for subsets of neurons, but not for glia, in monkey brains.
Department of Animal Models for Human Disease, National Institute of Neuroscience, NCNP, Tokyo, Japan.
Yoshito
Y
Masamizu
Takashi
T
Okada
Hidetoshi
H
Ishibashi
Shin'ichi
S
Takeda
Shigeki
S
Yuasa
Kiyoshi
K
Nakahara
D000818:Animals; D001370:Axonal Transport / Q000502:physiology; D001921:Brain / Q000378:metabolism* / Q000601:surgery*; D002144:Callithrix / Q000502:physiology* / Q000601:surgery*; D000229:Dependovirus / Q000235:genetics* / Q000378:metabolism; D018014:Gene Transfer Techniques / Q000592:standards*; D005822:Genetic Vectors / Q000235:genetics / Q000378:metabolism; D049452:Green Fluorescent Proteins / Q000235:genetics / Q000378:metabolism; D008297:Male; D011993:Recombinant Fusion Proteins / Q000235:genetics / Q000378:metabolism
D011993:Recombinant Fusion Proteins; C485184:enhanced green fluorescent protein; D049452:Green Fluorescent Proteins