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Gene delivery to cone photoreceptors by subretinal injection of rAAV2/6 in the mouse retina

Hori, Tesshu, Fukutome, Masashi, Maejima, Chiseto, Matsushima, Hiroki, Kobayashi, Kensuke, Kitazawa, Soichiro, Kitahara, Ryo, Kitano, Katsunori, Kobayashi, Kenta, Moritoh, Satoru, Koike, Chieko
Biochemical and biophysical research communications 2019 v.515 no.1 pp. 222-227
Dependoparvovirus, adults, capsid, cones (retina), gene therapy, gene transfer, mice, serotypes
Adeno-associated virus (AAV) has been studied as a safe delivery tool for gene therapy of retinal blinding diseases such as Leber's congenital amaurosis (LCA). The tropism of recombinant AAV (rAAV) including its specificity and efficiency in targeting retinal cell types has been studied with native or engineered capsids, along with specific promoters. However, one of the rAAV serotypes, rAAV2/6, has not been well-studied based on a report of low infection efficiency in the retina. We investigated the tropism of several rAAVs by subretinal injection in the adult mouse and found that rAAV2/6 predominantly infected cone photoreceptors including the main spectral type. Our data suggest that subretinal injection with rAAV2/6 may provide both an efficacious and specific means of gene delivery to cone photoreceptors in murine retinas.