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

Tesshu Hori; Masashi Fukutome; Chiseto Maejima; Hiroki Matsushima; Kensuke Kobayashi; Soichiro Kitazawa; Ryo Kitahara; Katsunori Kitano; Kenta Kobayashi; Satoru Moritoh; Chieko Koike

doi:10.1016/j.bbrc.2019.05.117

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

Tesshu Hori, Masashi Fukutome, Chiseto Maejima, Hiroki Matsushima, Kensuke Kobayashi, Soichiro Kitazawa, Ryo Kitahara, Katsunori Kitano, Kenta Kobayashi, Satoru Moritoh, Chieko Koike

Research output: Contribution to journal › Article › peer-review

Abstract

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.

Original language	English
Pages (from-to)	222-227
Number of pages	6
Journal	Biochemical and Biophysical Research Communications
Volume	515
Issue number	1
DOIs	https://doi.org/10.1016/j.bbrc.2019.05.117
Publication status	Published - 2019 Jul 12
Externally published	Yes

Keywords

Adeno-associated virus
Cone photoreceptors
Gene therapy
The mammalian retina

ASJC Scopus subject areas

Biophysics
Biochemistry
Molecular Biology
Cell Biology

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10.1016/j.bbrc.2019.05.117

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

abstract = "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.",

keywords = "Adeno-associated virus, Cone photoreceptors, Gene therapy, The mammalian retina",

author = "Tesshu Hori and Masashi Fukutome and Chiseto Maejima and Hiroki Matsushima and Kensuke Kobayashi and Soichiro Kitazawa and Ryo Kitahara and Katsunori Kitano and Kenta Kobayashi and Satoru Moritoh and Chieko Koike",

note = "Funding Information: We are indebted to Dr. Steven H. DeVries of Northwestern University Feinberg School of Medicine for helpful suggestions. This work was supported by Precursory Research for Embryonic Science and Technology from the Japan Science and Technology Agency (PRESTO, JST), by grants from the Ministry of Education program Grants-in-Aid for Scientific Research (B) ( JSPS KAKENHI grant number JP24390019 ), by the Industry to support private universities building up their foundations of strategic research from Ministry of Education, Culture, Sports, Science and Technology (MEXT grant number S1511027 ), by the Takeda Science Foundation and R-GIRO ( Ritsumeikan Global Innovation Research Organization, Ritsumeikan University ). Publisher Copyright: {\textcopyright} 2019",

year = "2019",

month = jul,

day = "12",

doi = "10.1016/j.bbrc.2019.05.117",

language = "English",

volume = "515",

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journal = "Biochemical and Biophysical Research Communications",

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T1 - Gene delivery to cone photoreceptors by subretinal injection of rAAV2/6 in the mouse retina

AU - Hori, Tesshu

AU - Fukutome, Masashi

AU - Maejima, Chiseto

AU - Matsushima, Hiroki

AU - Kobayashi, Kensuke

AU - Kitazawa, Soichiro

AU - Kitahara, Ryo

AU - Kitano, Katsunori

AU - Kobayashi, Kenta

AU - Moritoh, Satoru

AU - Koike, Chieko

N1 - Funding Information: We are indebted to Dr. Steven H. DeVries of Northwestern University Feinberg School of Medicine for helpful suggestions. This work was supported by Precursory Research for Embryonic Science and Technology from the Japan Science and Technology Agency (PRESTO, JST), by grants from the Ministry of Education program Grants-in-Aid for Scientific Research (B) ( JSPS KAKENHI grant number JP24390019 ), by the Industry to support private universities building up their foundations of strategic research from Ministry of Education, Culture, Sports, Science and Technology (MEXT grant number S1511027 ), by the Takeda Science Foundation and R-GIRO ( Ritsumeikan Global Innovation Research Organization, Ritsumeikan University ). Publisher Copyright: © 2019

PY - 2019/7/12

Y1 - 2019/7/12

N2 - 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.

AB - 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.

KW - Adeno-associated virus

KW - Cone photoreceptors

KW - Gene therapy

KW - The mammalian retina

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JO - Biochemical and Biophysical Research Communications

JF - Biochemical and Biophysical Research Communications

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ER -

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

Abstract

Keywords

ASJC Scopus subject areas

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