Targeting ON-bipolar cells by AAV gene therapy stably reverses LRIT3-congenital stationary night blindness

Adeno-associated virus (AAV)–based gene therapies aimed at curing inherited retinal diseases to date have typically focused on photoreceptors and retinal pigmented epithelia within the relatively accessible outer retina. However, therapeutic targeting in diseases such as congenital stationary night...

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Detalles Bibliográficos
Autores principales: Miyadera, Keiko, Santana, Evelyn, Roszak, Karolina, Iffrig, Sommer, Visel, Meike, Iwabe, Simone, Boyd, Ryan F., Bartoe, Joshua T., Sato, Yu, Gray, Alexa, Ripolles-Garcia, Ana, Dufour, Valérie L., Byrne, Leah C., Flannery, John G., Beltran, William A., Aguirre, Gustavo D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: National Academy of Sciences 2022
Materias:
Biological Sciences
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9060458/
https://www.ncbi.nlm.nih.gov/pubmed/35316139
http://dx.doi.org/10.1073/pnas.2117038119

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9060458/
https://www.ncbi.nlm.nih.gov/pubmed/35316139
http://dx.doi.org/10.1073/pnas.2117038119

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