Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

Usher 1 patients are born profoundly deaf and then develop retinal degeneration. Thus they are readily identified prior to the onset of retinal degeneration, making gene therapy a viable strategy to prevent their blindness. Here, we have investigated the use of adeno-associated viruses (AAV) for the...

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Main Authors: Lopes, Vanda S., Boye, Shannon E., Louie, Carrie M., Boye, Sanford, Dyka, Frank, Chiodo, Vince, Fofo, Hugo, Hauswirth, William W., Williams, David S.
Format: Artigo
Jezik:Inglês
Izdano: 2013
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Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC3640772/
https://ncbi.nlm.nih.gov/pubmed/23344065
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2013.3
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