Gene delivery to cone photoreceptors by subretinal injection of rAAV2/6 in the mouse retina
| Autor: | Masashi Fukutome, Chieko Koike, Ryo Kitahara, Tesshu Hori, Hiroki Matsushima, Katsunori Kitano, Satoru Moritoh, Kensuke Kobayashi, Chiseto Maejima, Soichiro Kitazawa, Kenta Kobayashi |
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| Rok vydání: | 2019 |
| Předmět: |
0301 basic medicine
Mice 129 Strain genetic structures viruses Genetic enhancement Genetic Vectors Leber Congenital Amaurosis Biophysics Biology Gene delivery Biochemistry Virus Retina law.invention Injections 03 medical and health sciences chemistry.chemical_compound 0302 clinical medicine Retinal Diseases law medicine Animals Molecular Biology Tropism Opsins Promoter Retinal Cell Biology Genetic Therapy Dependovirus eye diseases Cell biology 030104 developmental biology medicine.anatomical_structure Treatment Outcome chemistry 030220 oncology & carcinogenesis Recombinant DNA Retinal Cone Photoreceptor Cells sense organs |
| Zdroj: | Biochemical and biophysical research communications. 515(1) |
| ISSN: | 1090-2104 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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