Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness

Adeno-associated virus (AAV) effectively targets therapeutic genes to photoreceptors, pigment epithelia, Müller glia and ganglion cells of the retina. To date, no one has shown the ability to correct, with gene replacement, an inherent defect in bipolar cells (BCs), the excitatory interneurons of th...

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Publicado no:Hum Mol Genet
Main Authors: Scalabrino, Miranda L., Boye, Sanford L., Fransen, Kathryn M. H., Noel, Jennifer M., Dyka, Frank M., Min, Seok Hong, Ruan, Qing, De Leeuw, Charles N., Simpson, Elizabeth M., Gregg, Ronald G., McCall, Maureen A., Peachey, Neal S., Boye, Shannon E.
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2015
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Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4612567/
https://ncbi.nlm.nih.gov/pubmed/26310623
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv341
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