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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.
Afiliação
  • Hori T; Graduate School of Pharmacy, Ritsumeikan University, Kusatsu, Shiga, Japan; College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, Japan.
  • Fukutome M; College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, Japan; Graduate School of Life Sciences, Ritsumeikan University, Kusatsu, Shiga, Japan.
  • Maejima C; College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, Japan.
  • Matsushima H; Graduate School of Pharmacy, Ritsumeikan University, Kusatsu, Shiga, Japan; College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, Japan.
  • Kobayashi K; College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, Japan; Graduate School of Life Sciences, Ritsumeikan University, Kusatsu, Shiga, Japan.
  • Kitazawa S; College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, Japan.
  • Kitahara R; College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, Japan; Graduate School of Life Sciences, Ritsumeikan University, Kusatsu, Shiga, Japan.
  • Kitano K; College of Information Science and Engineering, Ritsumeikan University, Kusatsu, Shiga, Japan; Center for Systems Vision Science, Research Organization of Science and Technology, Ritsumeikan University, Kusatsu, Shiga, Japan.
  • Kobayashi K; Section of Viral Vector Development, National Institute for Physiological Sciences, Okazaki, Aichi, Japan.
  • Moritoh S; College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, Japan; Center for Systems Vision Science, Research Organization of Science and Technology, Ritsumeikan University, Kusatsu, Shiga, Japan.
  • Koike C; College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, Japan; Graduate School of Life Sciences, Ritsumeikan University, Kusatsu, Shiga, Japan; Center for Systems Vision Science, Research Organization of Science and Technology, Ritsumeikan University, Kusatsu, Shiga, Japan. Elect
Biochem Biophys Res Commun ; 515(1): 222-227, 2019 07 12.
Article em En | MEDLINE | ID: mdl-31146917
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.
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Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Retina / Doenças Retinianas / Terapia Genética / Células Fotorreceptoras Retinianas Cones / Dependovirus / Vetores Genéticos Limite: Animals Idioma: En Revista: Biochem Biophys Res Commun Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Japão
Texto completo
Adicionar na Minha BVS
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Retina / Doenças Retinianas / Terapia Genética / Células Fotorreceptoras Retinianas Cones / Dependovirus / Vetores Genéticos Limite: Animals Idioma: En Revista: Biochem Biophys Res Commun Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Japão